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SMA Foundation
11/20/2017

Central and Peripheral Treatment of SMA meeting summary

On November 10th, the SMA Foundation held a meeting with over 100 SMA researchers, clinicians, and patient advocacy group representatives to discuss the potential impact of SMN upregulation outside of the central nervous system (CNS). The meeting focused on evidence from preclinical models and patients that indicate SMN has a role outside of the CNS […]

Read more 4/24/2017

Are you a Type II/III SMA Patient or a Caregiver?

Help us create App games to measure SMA patients’ motor abilities and strength. Click here for more information.

Read more 12/29/2016

FDA Approves Biogen’s SPINRAZA™ (nusinersen) for Spinal Muscular Atrophy

The SMA Foundation is excited to share the news that the U.S. Food and Drug Administration (FDA) approved Biogen’s SPINRAZA™ (nusinersen) under Priority Review for the treatment of Spinal Muscular Atrophy (SMA) in pediatric and adult patients. SPINRAZA is the first and only treatment approved for SMA. (Please see http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm534611.htm for additional information.) SPINRAZA offers […]

Read more 12/1/2016

Comprehensive Book on Spinal Muscular Atrophy Published

We are pleased to announce that the book “Spinal Muscular Atrophy: Disease Mechanisms and Therapy,” has been published by Elsevier and is now available in retail outlets, as well as at the Elsevier Store in print and electronic formats here. This book is a comprehensive reference of recent advances in basic and clinical SMA research […]

Read more 1/7/2016

Spinal Muscular Atrophy Program Advances with Additional Product Candidate Entering Clinical Development

-Phase 1 Study Initiated in Healthy Volunteers with New SMN2 Splicing Modifier – RG7916 – SOUTH PLAINFIELD, N.J., Jan. 7, 2016 /PRNewswire/ — PTC Therapeutics, Inc. (Nasdaq: PTCT) today announced that RG7916, an additional SMN2 splicing modifier from the company’s joint development program with Roche and the SMA Foundation in spinal muscular atrophy (SMA), has […]

Read more 9/28/2015

Breaking News from the Muscular Dystrophy Association: Congress Passes the Ensuring Access to Clinical Trials Act (S. 139/H.R. 209)

This afternoon the U.S House of Representatives passed the Ensuring Access to Clinical Trials Act of 2015 (EACT) (S. 139/H.R. 209). Since the U.S. Senate passed the bill in July, the Act is now on its way to President Obama’s desk to be signed into law. EACT makes permanent a law that allows individuals to […]

Read more 3/13/2015

SMA Foundation Appoints Story Landis to its Board of Directors

The SMA Foundation has appointed Story Landis to its Board of Directors, effective February 24, 2015. “We are deeply honored to have Dr. Landis join the Foundation’s board. Her commitment to research, therapeutics development and patients is extraordinary,” said Loren Eng, the Foundation’s President. “We look forward to having Dr. Landis’ input and guidance in […]

Read more 12/30/2014

Every Life Foundation for Rare Diseases issues end of year action alert

As 2014 comes to a close, we are already preparing for what lies ahead for the rare disease community. Congress has pledged to make biomedical innovation a top-priority in 2015, and it is vital that we use this opportunity to usher in progress for rare disease patients.   I’m writing you today with two simple […]

Read more 12/19/2014

SMA Foundation comments to FDA on the importance of combination therapy for rare diseases like SMA

Last month, the FDA established a public docket for comments on FDA activities performed under the Food and Drug Administration Safety and Innovation Act (FDASIA), Patient Participation in Medical Product Discussions. The SMA Foundation took this opportunity to encourage the FDA to convene a stakeholder meeting on combination therapies, with the goal of producing a […]

Read more 8/7/2014

Orally delivered compounds, which selectively modify RNA splicing, prevent deficits in mouse models of Spinal Muscular Atrophy

Results published in Science show small molecule SMN2 splicing modifiers to be effective in SMA mouse models. Today the journal Science published results of a preclinical study demonstrating that treatment with orally available RNA splicing modifiers of the SMN2 gene starting early after birth prevents deficits in a mouse model of Spinal Muscular Atrophy (SMA). […]

Read more 10/11/2012

Congratulations to Dr. Brian McCabe and Dr. Livio Pellizzoni for Their Recent Publications in Cell

Congratulations to Dr. Brian McCabe and Dr. Livio Pellizzoni of Columbia University’s Motor Neuron Center for their publications in Cell. The first study, led by Dr. McCabe, shows that SMN restoration in proprioceptive neurons and interneurons corrects defects in motor neurons and muscles in a fly SMA model. Surprisingly, SMN restoration in muscles or motor […]

Read more 7/10/2012

President Obama Signs into Law The Food and Drug Administration Safety and Innovation Act (FDASIA)

On July 9, 2012, President Obama signed into law The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187. In their news release, the EveryLife Foundation for Rare Diseases highlights a key aspect of this legislation as pertinent to SMA drug development —  it will “empower [the] FDA to use all the science […]

Read more 5/4/2012

The Spinal Muscular Atrophy Foundation Announces the Publication of Results from the Biomarkers for SMA (BforSMA) Clinical Study

NEW YORK, NY | May 4, 2012 – The Spinal Muscular Atrophy (SMA) Foundation congratulates the members of the Pilot Study of Biomarkers for Spinal Muscular Atrophy (BforSMA) Trial Group for the recent publication of study results in two articles published by the open-access, peer-reviewed journal, PLoS ONE. The goal of the BforSMA study was […]

Read more 4/3/2012

The Spinal Muscular Atrophy Foundation Announces a Biomarker Panel to Guide SMA Therapeutic Development

NEW YORK, NY | April 3, 2012 – The Spinal Muscular Atrophy (SMA) Foundation announced today the launch of a biomarker assay panel for SMA using Myriad RBM’s Multi-Analyte Profiling (MAP) technology platform. The SMA-MAP panel is designed to evaluate the severity of SMA and disease progression and can be used to assess drug efficacy […]

Read more 3/9/2012

NORD Supports FAST Act Introduced by Representatives Stearns and Towns

Two bills have been introduced in Congress which are intended to expedite the drug approval process for diseases with serious unmet medical need such as SMA. On February 15, 2012, the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act, S. 2113, was introduced by Senator Kay Hagan (D-NC) and on March 5, 2012 […]

Read more 2/21/2012

SMA Foundation Observes Rare Disease Day

New York, NY, February 21, 2012 – The Spinal Muscular Atrophy (SMA) Foundation will join the National Organization for Rare Disorders (NORD) and others around the world in observing Rare Disease Day on February 29.  The purpose is to focus attention on the needs of patients and families affected by rare diseases. “This is a […]

Read more 2/13/2012

Roche, PTC & SMA Foundation Agreement to Develop a Treatment for SMA Receives Extensive Coverage

On November 29, 2011, Roche, PTC Therapeutics and the SMA Foundation announced the signing of an agreement to develop a treatment for Spinal Muscular Atrophy. Several media sources reported on the agreement including BioCentury, BioWorld Today, Bloomberg Businessweek, Chemical & Engineering News, Clinical Professionals, Dow Jones VentureWire, Drug Discovery News, FierceBiotech, Genetic Engineering and Biology News, […]

Read more 12/19/2011

Isis Initiates Phase 1 Clinical Study of ISIS-SMNRx in Patients with Spinal Muscular Atrophy

CARLSBAD, Calif., December 19, 2011 – Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has initiated a Phase 1 study of ISIS-SMNRx in patients with spinal muscular atrophy (SMA). SMA is a severe motor-neuron disease that is the leading genetic cause of infant mortality. Isis is developing ISIS-SMNRx as a potential treatment for all […]

Read more 11/29/2011

Roche Signs Agreement with PTC Therapeutics to Advance Treatment for Spinal Muscular Atrophy (SMA)

Collaboration offers new hope for a potential treatment for the leading genetic cause of death in infants and toddlers Basel, Switzerland, South Plainfield, NJ, USA and New York, NY, USA – November 29, 2011 – Roche (SIX: RO, ROG; OTCQX: RHHBY), PTC Therapeutics, Inc. (PTC) and the SMA Foundation, announced today a licensing agreement for […]

Read more 11/23/2011

Congratulations to Stephen Mikita, Esq., on his new role as a Patient Representative to the FDA

Congratulations to SMA Foundation Board Member, Stephen Mikita, Esq., on his new role as a Patient Representative to the U.S. Food and Drug Administration (FDA). Mr. Mikita recently completed the Patient Representative training program with the FDA’s Office of Special Health Issues. As a Patient Representative, Mr. Mikita will participate in advisory committees to review […]

Read more 9/9/2011

Save the Date – SfN 2011

Save the Date – Society for Neuroscience 2011 Monday November 14, 2011 6:30 – 9:30 p.m. Washington Convention Center, 150B Pretzels and endplates: Motor neuron pathology and the role of SMN in motor neuron development Chien-Ping Ko, PhD, University of Southern California “Synaptic defects in the spinal and neuromuscular circuitry in SMA” George Mentis, PhD, […]

Read more 9/8/2011

Promotions and Staff Additions at the SMA Foundation

The SMA Foundation announced today that Karen Chen, PhD, has been named Chief Operating Officer (COO). In addition to Dr. Chen’s continued role as Chief Scientific Officer (CSO), she will also be responsible for managing the day-to-day business operations of the Foundation. Additionally, both Dione Kobayashi, PhD and Sergey Paushkin, MD, PhD have been promoted […]

Read more 7/27/2011

Increasing SMN Protein May Be Therapeutic Even After SMA Disease Onset

Congratulations to Umrao Monani, Assistant Professor at the Motor Neuron Center of Columbia University and Cathleen Lutz, Associate Director of Genetic Research Science at The Jackson Laboratory, and their colleagues for their new publication “Postsymptomatic Restoration of SMN Rescues the Disease Phenotype in a Mouse Model of Severe Spinal Muscular Atrophy” in the Journal of […]

Read more 6/27/2011

Congratulations to Dr. Lee Rubin for His Recent Publication in Nature Chemical Biology

Congratulations to SMA Foundation Investigator, Lee Rubin, Director of Translational Medicine at the Harvard Stem Cell Institute, and his team for publishing their article, “A Screen for Regulators of Survival of Motor Neuron Protein Levels” in Nature Chemical Biology on June 19, 2011. The article describes a novel image-based screen to identify compounds that increase […]

Read more 3/15/2011

Enzo Biochem Unit Launches First-to-Market Survival Motor Neuron (SMN) Protein Immunoassay System

FARMINGDALE, NY and NEW YORK, NY – March 15, 2011 – Enzo Biochem, Inc. and the Spinal Muscular Atrophy (SMA) Foundation today announced that Enzo’s wholly owned subsidiary, Enzo Life Sciences Inc., has launched a unique immunoassay (ELISA) system which can be used for the identification and detection of Survival Motor Neuron (SMN) protein. The kit […]

Read more 3/11/2011

Cynthia Joyce Elected Vice-Chair of the SMA Foundation Board of Directors

The SMA Foundation announced today that Cynthia Joyce will be stepping down as Executive Director of the Foundation at the end of this month. Effective April 2, 2011, Ms. Joyce will become a member of the Foundation’s Board of Directors and has been elected to the office of Vice-Chair. Ms. Joyce was the first employee […]

Read more 2/11/2011

NY State Dept. of Health Announces Genetic Diseases of Children Conference – March 8-9, 2011 in NYC

The New York State Department of Health Announces Genetic Diseases of Children…Advancing Research & Care Conference to be held on March 8-9, 2011 at the Sheraton New York Towers & Hotel in New York City. This is a national conference focused on creating opportunities to advance research and improve the delivery of health care for […]

Read more 2/10/2011

Congratulations to Dr. George Mentis for His Recent Publication in Neuron

Congratulations to SMA Foundation Investigator, George Mentis, Assistant Professor at the Motor Neuron Center of Columbia University, for his recent article “Early Functional Impairment of Sensory-Motor Connectivity in a Mouse Model of Spinal Muscular Atrophy” published in Neuron on February 10, 2011. The article examines the onset of motor neuron loss in a mouse model […]

Read more 2/10/2011

Dr. Seward Rutkove Receives $1MM Biomarker Prize from the Prize4Life Foundation

Congratulations to Foundation Investigator Seward Rutkove, the recipient of the $1MM Biomarker Prize from the Prize4Life Foundation! The Prize was awarded for Dr. Rutkove’s work to advance the use of electrical impedance myography (EIM) as a biomarker measure for ALS. The SMA Foundation funded a pilot study of EIM in children with SMA to determine […]

Read more 1/20/2011

Experienced leaders in business, media, and law, join the SMA Foundation’s Board of Directors

New York, NY – January 20, 2011 – The Spinal Muscular Atrophy Foundation announces the addition of Andrew Knight, Helen Meates, Stephen Mikita, Juli Oh and Simon Prisk to the SMA Foundation Board of Directors. They join a distinguished group of business and scientific leaders who oversee the SMA Foundation. Currently, there is no treatment […]

Read more 1/19/2011

SMA Foundation and RBM Collaborate on Biomarker Panels to Guide SMA Therapeutic Development

NEW YORK, NY AND AUSTIN, TX – January 19, 2011 – The Spinal Muscular Atrophy (SMA) Foundation and Rules-Based Medicine, Inc. (RBM) announced today that they have reached the first milestone in a program to develop a panel of plasma protein biomarkers for SMA using RBM’s Multi-Analyte Profiling (MAP) technology platform. In this collaboration, RBM […]

Read more 12/16/2010

Exercise Study News Item: Recruiting for an Exercise and Strengthening Study in SMA Type III

Are you between the ages of 8 and 50 and diagnosed with SMA Type III? Are you interested in exercise and the effect it has on your body? The Columbia University SMA Clinical Research Center is conducting an 18 month trial of the effects of cardiovascular and strengthening exercise on individuals with SMA Type III. […]

Read more 12/15/2010

Pre-Application Meeting for the Network of Excellence in Neuroscience Clinical Trials (NEXT)

Notice Number: NOT-NS-11-005 Key Dates Release Date: December 7, 2010 Issued by National Institute of Neurological Disorders and Stroke (NINDS) Purpose The National Institute of Neurological Disorders and Stroke (NINDS) will hold a pre-application meeting regarding the Request for Applications (RFA) for Clinical Research Sites for the Network of Excellence in Neuroscience Clinical Trials (RFA-NS-11-008), […]

Read more 10/26/2010

NIH Conference and Legislative Update

Dear Families, Thank you for all you have done to increase awareness and attention towards Spinal Muscular Atrophy (SMA). As most of you are aware, this has been an eventful year in the U.S. Congress; national health care and research priorities have been prominent on the radar screens of lawmakers. The significant visibility that you […]

Read more 8/6/2010

SMA Foundation and Enzo Life Sciences Inc. Partner to Release New SMN EIA Kit

The Spinal Muscular Atrophy (SMA) Foundation is pleased to announce that Enzo Life Sciences Inc. has become its lead partner in the development and distribution of a limited quantity of newly developed assay kits designed to measure Survival Motor Neuron (SMN) protein, the critical protein that is deficient in Spinal Muscular Atrophy. The kit is […]

Read more 5/25/2010

SMA Patients Look to Interdisciplinary CUMC Team for Solutions to Everyday Challenges

Working with Columbia Technology Ventures, a team of individuals ranging from professors at Columbia University College of Physicians and Surgeons to undergraduate students at Columbia’s School of Engineering and Applied Science has developed a device meant to improve arm function in patients with SMA. The GNO is a sling-like mechanism that may make it possible […]

Read more 5/5/2010

SMA Foundation and Assay Designs, Inc. Release New SMN EIA Kit

The Spinal Muscular Atrophy (SMA) Foundation and Assay Designs, Inc. are pleased to announce the release of a limited quantity of newly developed assay kits designed to measure Survival Motor Neuron (SMN) protein, the critical protein that is deficient in Spinal Muscular Atrophy. The Assay Designs® SMN (human) Enzyme Immunometric Assay (EIA) kit is a […]

Read more 4/20/2010

Help with Research – Coriell Institute for Medical Research Is Seeking SMA Blood Sample Donations

The Coriell Institute for Medical Research is a not-for-profit research organization “dedicated to understanding human genetic diseases and providing the highest quality genetic resources”. Coriell houses the world’s largest biorepository of cell cultures and offers medical researchers from around the world access to these patient cell lines for study of disease genes, use in screening […]

Read more 3/25/2010

SMA Foundation Announces the Discovery of Over 400 Candidate Biomarkers from the BforSMA Study

NEW YORK, NY – March 25, 2010 – The Spinal Muscular Atrophy (SMA) Foundation, a nonprofit organization dedicated to accelerating the development of a treatment for SMA, is pleased to announce the completion of a pilot study to identify Biomarkers for Spinal Muscular Atrophy (BforSMA) and the discovery of over 400 candidate biomarkers. Next steps […]

Read more 10/27/2009

Families of SMA and Repligen Corporation

The SMA Foundation congratulates Families of SMA and Repligen Corporation on the announcement of a licensing deal for the development of new compounds for the treatment of SMA. This marks a welcome new stage of advancement in SMA therapeutics development – and a clear demonstration of the value of non-profit drug development activities. For more […]

Read more 5/28/2009

Dr. Hung Li

We note with sadness the passing of Dr. Hung Li, a pioneer in Spinal Muscular Atrophy research, in March of this year. Dr. Li was passionate about SMA basic and therapeutic research, committed to high-quality science and doing the right thing for SMA patients and families. His contributions to the field include the introduction of […]

Read more 5/27/2009

SMA Foundation Announces Completion of Biomarker Study Enrollment

NEW YORK, NY – March 27, 2009 – The Spinal Muscular Atrophy (SMA) Foundation, a nonprofit organization dedicated to accelerating the development of a treatment for SMA, is pleased to announce the completion of enrollment for the pilot study of Biomarkers for Spinal Muscular Atrophy (BforSMA). The first subject was enrolled in the BforSMA study […]

Read more 5/12/2009

PTC Therapeutics and Spinal Muscular Atrophy Foundation Announce $8.5 million Small Molecule Development Collaboration

SOUTH PLAINFIELD, NJ AND NEW YORK, NY – May 12, 2009 – PTC Therapeutics, Inc. (PTC) and the Spinal Muscular Atrophy (SMA) Foundation today announced the expansion of their research collaboration in which the SMA Foundation will provide up to $8.5 million in funding to PTC. This is the second continuation of the collaboration to fund […]

Read more 1/29/2009

Biomarker Study Recruitment Update

The Biomarkers for Spinal Muscular Atrophy (BforSMA) clinical trial is a current pilot study looking to identify potential biomarkers (measures) that can be used to evaluate SMA disease severity and future treatments. Recruitment is ongoing and as of January 29, 2009, 71 subjects have been recruited from 18 clinical sites in the United States and […]

Read more 10/27/2008

Social Security Announces Nationwide Launch of Compassionate Allowances

Michael J. Astrue, Commissioner of Social Security, today announced the national rollout of the agency’s Compassionate Allowances initiative, a way to expedite the processing of disability claims for applicants whose medical conditions are so severe that their conditions obviously meet Social Security’s standards. For a full list of 50 conditions that have been selected for […]

Read more 10/24/2008

SMA Foundation Announces a Pilot Study of Biomarkers for SMA

NEW YORK, NY – October 24, 2008 – The Spinal Muscular Atrophy (SMA) Foundation, a nonprofit organization dedicated to accelerating the development of a treatment for SMA, is pleased to announce the launch of a pilot study of Biomarkers for Spinal Muscular Atrophy (BforSMA). The goal of this study is to identify a potential biomarker […]

Read more 10/15/2008

SMA Patient Registry New Website is Here!

SMA Patient Registry New Website is Here! https://smaregistry.iu.edu/ Indianapolis, IN —October 15, 2008 – After many months of anticipation we are pleased to announce the unveiling of the new International Spinal Muscular Atrophy Patient Registry Website! The Website now allows individuals to participate in the Registry completely through on-line interaction including registering, completing questionnaires, learning about and […]

Read more 9/17/2008

Assay Designs, Inc. and the Spinal Muscular Atrophy Foundation announce collaboration

ANN ARBOR, Mich. — Sept. 15, 2008 – Assay Designs and the Spinal Muscular Atrophy Foundation (SMAF) are very pleased to announce a collaborative agreement for development of reagents and assays for SMN (Survival Motor Neuron) protein to expedite drug discovery and development efforts for spinal muscular atrophy (SMA), the leading genetic cause of mortality in […]

Read more 4/25/2008

SMA Foundation to Receive Contributions from Build-A-Bear Workshop Fundraising Effort

ST. LOUIS — APRIL 25, 2008 – Today Build-A-Bear Workshop® introduced a new member to its Bearemy’s Kennel Pals® line – the collection of plush dogs that benefit domestic pet programs across the United States. The latest addition, the Black Labrador, was inspired by the story of a beary special Guest, Shea Megale, and her friendship […]

Read more 4/24/2008

Groups Release Family Version of SMA Care Standard

The Patient Advisory Group of the International Coordinating Committee (ICC) for SMA Clinical Trials has published a family-friendly set of guidelines for care in spinal muscular atrophy (SMA) – click here to download the guide – to complement the physician guidelines published in August (for more on the physicians’ consensus statement, see The Journal of […]

Read more 12/6/2007

PTC Therapeutics Announces Additional $1.6 Million Grant from the SMA Foundation

SOUTH PLAINFIELD, NJ – December 6, 2007 – PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of small-molecule drugs targeting post-transcriptional control mechanisms, today announced an expanded research collaboration with the Spinal Muscular Atrophy (SMA) Foundation. The collaboration builds on the existing research agreement to identify and characterize compounds that […]

Read more 11/6/2007

SMA Summit on Drug Development in Neurology Today

Neurology Today: SMA Drug Development Gains Momentum Under NINDS Model for Neuro-Research Partnerships. Reprinted with permission from the American Academy of Neurology from Neurology Today, Nov. 6, 2007.

Read more 10/2/2007

Raising Awareness for Children with SMA

The SMA Foundation warmly thank Megan and Shea Megale for all their efforts in raising awareness for children living with SMA. For more information about a recent book signing event in New York City, please see this article in USA Today and the Megales’ interview on the Today show.

Read more 9/8/2007

SMA Foundation and BG Medicine Announce Collaboration

New York, NY and Waltham, MA – September 18, 2007 – The Spinal Muscular Atrophy Foundation and BG Medicine today announced a collaboration to discover plasma biomarkers of drug efficacy for spinal muscular atrophy (SMA), the leading genetic cause of mortality in infants and toddlers. This project seeks to discover a clinically-useful molecular biomarker, which can […]

Read more 3/13/2007

CSHL Shows Correcting RNA Splicing May Help Treat SMA

March 13, 2007 — Cold Spring Harbor, New York – RNA splicing antisense technology studied at Cold Spring Harbor Laboratory (CSHL) effectively corrected an mRNA splicing defect found in spinal muscular atrophy (SMA) patients, and is now ready to be tested in mouse models. “SMA patients who suffer from motor-neuron degeneration may benefit from our […]

Read more 3/2/2007

SMA Investigator’s Research Highlighted in Science

Spinal muscular atrophy (SMA) is an inherited disease characterized by the selective death of motor neurons, resulting in generalized muscle weakness that is often fatal in infancy or early childhood. SMA is caused by deletions or mutations in the gene encoding survival motor neuron 1 protein (SMN1), whose function is unclear. SMN1 contributes to the […]

Read more 1/11/2007

SMA Investigator Receives 2006 MacArthur ‘Genius’ Award

MCB is pleased to announce that Kevin Eggan, Assistant Professor of Molecular and Cellular Biology is a recipient of this year’s MacArthur “genius” award in recognition of his stellar contribution to stem cell research. Eggan expressed his gratitude and appreciation for the award: “Needless to say, I was shocked, surprised, overwhelmed, the whole spectrum of […]

Read more 12/1/2006

Industry Leaders Join SMA Foundation’s Scientific Advisory Board

New York, NY – December 1, 2006 – The Spinal Muscular Atrophy Foundation announces the addition of Ivan Lieberburg, Ph.D., M.D. and Edward Kaye, M.D. to its Scientific Advisory Board. They join a distinguished group of business and scientific leaders who help formulate and execute the SMA Foundation’s drug discovery programs. Currently, there is no […]

Read more 5/11/2006

Lexicon Genetics Awarded Grant from the United States Army for Study of SMA

The Woodlands, Texas — May 11, 2006 – Lexicon Genetics Incorporated (Nasdaq: LEXG) announced today that it was awarded a grant from the United States Army Medical Research & Materiel Command (USAMRMC) for the identification of targets that may be important in the development of drugs to prevent or treat spinal muscular atrophy (SMA), a neurodegenerative […]

Read more 5/6/2006

SMA is Model for New Approaches to Orphan Drug Development

WASHINGTON, DC – May 16, 2010 – At a time when the cost of developing a new drug is skyrocketing and research money is tight, it is important to be creative in designing new therapies for rare diseases and bringing them to market, according to speakers at the eighth Annual Meeting of the American Society for […]

Read more 2/24/2006

PsychoGenics and SMA Foundation Extend Research Agreement

February 24, 2006 – PsychoGenics Inc. Tarrytown, New York and the Spinal Muscular Atrophy (SMA) Foundation New York, NY – have extended their research agreement to establish and behaviorally phenotype transgenic in vivo models of SMA and test potential therapeutics using these models. As part of this collaborative agreement PsychoGenics has established self-sustaining transgenic SMA colonies […]

Read more 1/31/2006

Critical Research Tools for SMA Drug Discovery

New York, NY — January 31, 2006 – The Spinal Muscular Atrophy Foundation announced the completion of a new licensing agreement that will facilitate access to intellectual property fundamental to drug discovery efforts for the disease. Spinal muscular atrophy (SMA) is the leading genetic killer of infants and toddlers; there is no known treatment or cure. […]

Read more 10/4/2005

The Spinal Muscular Atrophy Foundation Launches Collaboration for New Disease Models

New York, NY – October 4, 2005 – The Spinal Muscular Atrophy Foundation announced an agreement today with Regeneron Pharmaceuticals, Inc. to develop new animal models of spinal muscular atrophy (SMA), the leading genetic cause of mortality in infants and toddlers.. The collaboration will exploit Regeneron’s proprietary VelocigeneTM technology to rapidly generate and test new […]

Read more 5/17/2005

New Advisors Bring Diversity and Experience to SMA Foundation

New York, NY – May 17, 2005 – The Spinal Muscular Atrophy Foundation today announced the addition of six members to its Scientific Advisory Board. The new advisors, some of the most wellrespected individuals in their fields, were specifically sought for the wealth of experience they bring to bear on Foundation efforts to advance drug […]

Read more 3/3/2005

Prominent Researchers at Columbia University Receive Significant Funding to Combat SMA

New York, NY – March 03, 2005 – Researchers at Columbia University have received more than $3 million in funding from the Spinal Muscular Atrophy Foundation to conduct research on spinal muscular atrophy (SMA), a neuromuscular disease that is the leading genetic killer of infants and toddlers. The funding will enable the institution to significantly expand […]

Read more 1/31/2005

University of Wisconsin-Madison Scientists Create Spinal Motor Neurons from Embryonic Stem Cells

by Paroma Basu Scientists grow critical nerve cells MADISON, WI — January 31, 2005 – After years of trial and error, scientists have coaxed human embryonic stem cells to become spinal motor neurons, critical nervous system pathways that relay messages from the brain to the rest of the body. The new findings, reported online Jan. 30 in […]

Read more 11/29/2004

Columbia Researchers Identify Drug as Therapeutic Candidate for SMA

New York, NY  – November 29, 2004 – Using a newly developed technology, a team of Columbia University researchers has uncovered that indoprofen, a nonsteroidal anti-inflammatory drug, may increase the production of a protein lacking in patients with spinal muscular atrophy (SMA), a fatal pediatric genetic disease. It is the scientists’ hope that the discovery will […]

Read more 11/29/2004

SMA Foundation Funds Over $15 Million to Develop Treatments for SMA

New York, NY – November 29, 2004 – The Spinal Muscular Atrophy Foundation, a nonprofit organization dedicated to finding a treatment or cure for spinal muscular atrophy (SMA) – the leading genetic killer of infants and toddlers – announced that is has funded more than $15 million in research in the last 18 months. Projects were […]

Read more 10/21/2004

The Jackson Laboratory to Distribute Mouse Models for SMA

Bar Harbor, Maine – October 21, 2004 – The Jackson Laboratory is pleased to announce that it has received support from the Spinal Muscular Atrophy Foundation to make available the first group of mouse models for spinal muscular atrophy (SMA), a neuromuscular disease and the leading genetic cause of death among infants and toddlers. The […]

Read more 9/24/2004

Leading Medical Institutions Establish a Clinical Research Network to Study SMA

New York, NY – September 24, 2004 – Researchers at Columbia University, Children’s Hospital Boston and The Children’s Hospital of Philadelphia have established a clinical research network to study spinal muscular atrophy (SMA), the leading genetic killer of infants and toddlers. The Pediatric Neuromuscular Clinical Research Network will address the vital need in SMA research to […]

Read more 9/13/2004

CombinatoRx Inks R&D Deal With Foundation

CombinatoRx Inc. of Boston has inked a multimillion-dollar, two-year research and development deal with a foundation to find treatments for spinal muscular atrophy, a neuromuscular disease. The Boston startup said its deal with the New York-based Spinal Muscular Atrophy Foundation calls for two years of research and development funding from the foundation. CombinatoRx will also […]

Read more 9/7/2004

Curis Lands $5.4M Spinal Muscular Atrophy Grant

Curis Inc., a Cambridge-based drug developer, received a $5.4 million three-year grant to help treat spinal muscular atrophy, the company announced Tuesday. The Spinal Muscular Atrophy Foundation awarded the grant to Curis to identify therapeutic compounds that could be used to treat the neuromuscular disease, which is the leading genetic cause of infant and toddler […]

Read more 9/7/2004

Curis Awarded $5.4M to Identify Treatment for Spinal Muscular Atrophy

Curis Inc. in Cambridge has received a $5.4 million, three-year grant from the Spinal Muscular Atrophy Foundation to identify therapeutic compounds to treat spinal muscular atrophy, a debilitating neuromuscular disease that is the leading genetic cause of infant and toddler death. The study will use proprietary Curis technologies and expertise to develop and refine motor […]

Read more 4/19/2004

The AAN Foundation and the SMA Foundation Announce the Recipients of the 2004 Young Investigator Award in SMA

New York, NY – April 19, 2004 – The Spinal Muscular Atrophy Foundation today announced that it has awarded new research grants totaling $1.125 million to four outstanding Young Investigators specializing in research on Spinal Muscular Atrophy (SMA), the leading genetic killer of infants and toddlers. These grants support the implementation and advancement of clinical research […]

Read more 1/1/1970

Assay Designs, Inc. and the Spinal Muscular Atrophy Foundation announce collaboration

ANN ARBOR, Mich. — Sept. 15, 2008 – Assay Designs and the Spinal Muscular Atrophy Foundation (SMAF) are very pleased to announce a collaborative agreement for development of reagents and assays for SMN (Survival Motor Neuron) protein to expedite drug discovery and development efforts for spinal muscular atrophy (SMA), the leading genetic cause of mortality in […]

Read more