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04-May-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 to [...]

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 and [...]

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 [...]

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 [...]

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, [...]

19-Dec-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 [...]

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 [...]

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 [...]

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, [...]

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 [...]

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 [...]

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 [...]

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 [...]

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 [...]

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 [...]

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 [...]

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 [...]

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 [...]

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 [...]

16-Dec-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. [...]

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), [...]

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 [...]

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 [...]

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 [...]

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 [...]

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 [...]

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 [...]

27-Oct-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 [...]

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 [...]

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 [...]

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 [...]

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 [...]