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1/16/2018

AveXis Announces Expanded Clinical Development Program for AVXS-101in Spinal Muscular Atrophy

Chicago, Ill. (January 16, 2018)- AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an overview of the expanded clinical development program for the company’s initial gene therapy candidate, AVXS-101, for the treatment of spinal muscular atrophy (SMA). In addition to […]

Read more 12/13/2017

AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101

Chicago, Ill. (December 13, 2017) – AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has notified the company that, based on review of data submitted, the company may initiate its planned Phase 1 […]

Read more 10/31/2017

Ionis and Biogen Win Prestigious Prix Galien Best Biotechnology Product Award for SPINRAZA

The SMA Foundation congratulates Ionis and Biogen for being awarded the Prix Galien USA Award for the Best Biotechnology Product in 2017 for SPINRAZA® (nusinersen). The Prix Galien USA Award recognizes extraordinary achievement in scientific innovation that improves the state of human health. The award was presented at a ceremony in New York City on […]

Read more 9/29/2017

Novartis to resume enrollment in ongoing study of branaplam in type 1 SMA

Effective immediately, following the respective Health Authorities and Ethics Committee approvals, Novartis will be resuming enrollment in the ongoing study in type 1 SMA (CLMI070X2201) at their existing sites in Belgium, Germany, Denmark and Italy. Please click here to read the full letter.

Read more 9/29/2017

AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process

Chicago, Ill. (September 29, 2017) – AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has notified the company that based on review of data submitted, including the potency assay, it may initiate its […]

Read more 9/25/2017

Roche to begin Part 2 of the SUNFISH Trial

To read the full press release, please click here: SUNFISH

Read more 6/28/2017

Scholar Rock to Develop SRK-015 to Improve Muscle Function in Patients with Spinal Muscular Atrophy

First data on inhibition of myostatin activation in a preclinical model of SMA to be highlighted at the Cure SMA Annual Conference Treatment with SRK-015 resulted in significantly increased muscle strength in a genetic model of SMA CAMBRIDGE, Mass., June 28, 2017 – Scholar Rock, a biotechnology company focused on discovering and developing drugs that […]

Read more 1/6/2017

RG7916 Granted Orphan Drug Designation in the U.S. for the Treatment of Spinal Muscular Atrophy

SOUTH PLAINFIELD, N.J., January 6, 2017 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (OOD) to RG7916 for the treatment of patients with Spinal Muscular Atrophy (SMA). RG7916 is part of PTC’s joint development program in SMA with Roche and the SMA […]

Read more 1/6/2017

PTC Therapeutics Announces Initiation of FIREFISH Study in Infant (Type I) SMA Patients

SOUTH PLAINFIELD, N.J., January 5, 2017 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation (SMAF) initiated a clinical study in infants with Type I SMA. The study, named FIREFISH, will investigate the safety, tolerability and efficacy of RG7916 in […]

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 11/2/2016

AveXis Announces Single-Arm Design for U.S. Pivotal Study of AVXS-101 in SMA Type 1 Patients

CHICAGO, Nov. 01, 2016 (GLOBE NEWSWIRE) — AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that the planned pivotal study of AVXS-101 in spinal muscular atrophy (SMA) Type 1 will reflect a single-arm design, using natural history of the disease as […]

Read more 10/28/2016

Biogen’s Regulatory Applications for Nusinersen as a Treatment for Spinal Muscular Atrophy Accepted by FDA and EMA

Biogen today announced that its New Drug Application (NDA) for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), has been accepted by the U.S. Food and Drug Administration (FDA) for Priority Review, and that the company’s Marketing Authorization Application (MAA) has been validated by the European Medicines Agency (EMA). Nusinersen had previously been granted […]

Read more 9/27/2016

Biogen Submits NDA to the FDA for Nusinersen

Biogen and Ionis have taken a crucial step in the pathway to approval of nusinersen. Biogen has completed the submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) and will submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the coming weeks. Biogen has also applied […]

Read more 9/27/2016

Roche Initiates New Phase 2 Clinical Trials

Roche has announced that the company is initiating two new Phase 2 studies in patients with Spinal Muscular Atrophy (SMA) to evaluate RG7916—SUNFISH and FIREFISH. RG7916 is an orally available drug that directly targets the underlying molecular deficiency of SMA by modulating SMN2 splicing to increase expression of full-length SMN2 mRNA and SMN protein from […]

Read more 9/2/2016

Bloomberg Article: New Hope for Kids With a Rare Disease, Thanks to a Dinner Party

Since grade school, Lauren Gibbs had been a dogged defensive player on her wheelchair-basketball team. Offense wasn’t a big part of her game because it was a struggle to heave the ball high enough to hit the rim. Then Gibbs, who was born with spinal muscular atrophy, enrolled in a trial of an experimental drug […]

Read more 8/1/2016

Biogen and Ionis Pharmaceuticals Report Nusinersen Meets Primary Endpoint at Interim Analysis of Phase 3 ENDEAR Study in Infantile-Onset Spinal Muscular Atrophy

CAMBRIDGE, Mass. & CARLSBAD, Calif.–Aug. 1, 2016– Biogen (NASDAQ:BIIB) and Ionis Pharmaceuticals (NASDAQ:IONS) today announced that nusinersen, their investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint pre-specified for the interim analysis of ENDEAR, the Phase 3 trial evaluating nusinersen in infantile-onset (consistent with Type 1) SMA. The analysis found that infants receiving […]

Read more 7/25/2016

Update on LMI070 for the Treatment of Type 1 SMA

In May, we shared with you news of the difficult decision to pause enrollment for our study of LMI070 for the treatment of Type 1 Spinal Muscular Atrophy (SMA). This decision was made because results from an animal study, using daily dosing for a year compared to weekly dosing in the human study, showed unexpected […]

Read more 7/20/2016

AveXis Receives U.S. FDA Breakthrough Therapy Designation for AVXS-101 Gene Replacement Therapy for SMA Type 1

Chicago,Ill., July 20, 2016 – AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for AVXS-101, the company’s lead development candidate for the treatment of spinal muscular atrophy (SMA) Type 1 […]

Read more 6/1/2016

Letter to the SMA Community on the Study of LMI070 for the Treatment of Type 1 SMA

Wednesday May 25th, 2016 – Letter from Novartis: We have made the difficult decision to pause enrollment for our study of LMI070 for the treatment of Type 1 Spinal Muscular Atrophy (SMA). Animal safety studies were taking place in parallel with this trial, which is a common practice when studying potentially life threatening diseases to […]

Read more 6/1/2016

FightSMA Announces First-Time Inclusion of Spinal Muscular Atrophy in Defense Department’s Peer-Reviewed Medical Research Program

WASHINGTON, D.C., May 26, 2016–FightSMA is proud to announce that, for the first time, the U.S. Senate Appropriations committee has included spinal muscular atrophy as a research topic in the Defense Department’s Peer-Reviewed Medical Research Program as part of the Fiscal Year 2017 Defense Appropriations bill. This exciting news comes as the result of months […]

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 10/5/2015

Data from Ongoing Study of AVXS-­101 in Spinal Muscular Atrophy Type 1 Presented at World Muscle Congress

Brighton, United Kingdom. October 5, 2015 – AveXis, Inc., a clinical stage gene therapy company developing treatments for rare and life-­threatening neurological genetic diseases, presented data from its ongoing Phase 1/2 trial of AVXS-­101, the company’s initial product candidate for the treatment of spinal muscular atrophy (SMA) Type 1, at the International Congress of the […]

Read more 6/11/2015

ISIS PHARMACEUTICALS REPORTS DATA FROM ISIS-SMNRX PHASE 2 STUDY IN INFANTS WITH SPINAL MUSCULAR ATROPHY

Webcast to review study data scheduled for Thursday, June 11 at 8:30 a.m. Eastern Time CARLSBAD, Calif., June 11, 2015 – Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) today provided an update on its ongoing open-label Phase 2 clinical study of ISIS-SMNRx in infants with Type I spinal muscular atrophy (SMA). Previously the company reported data from […]

Read more 5/5/2015

Letter to the SMA community about the clinical development of RG7800

Dear Spinal Muscular Atrophy Community Members, We would like to update you on the clinical development of RG7800, an investigational SMN2 splicing modifier that is being studied as a potential treatment for spinal muscular atrophy (SMA). This compound is the result of a collaboration between Roche, PTC Therapeutics, and the SMA Foundation. In November 2014, […]

Read more 4/20/2015

Phase 1 data from Spinal Muscular Atrophy Program to be presented at the 2015 AAN Annual Meeting

WASHINGTON, D.C. – April 20, 2015 – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Phase 1 clinical data from the company’s joint development program with Roche and the SMA Foundation in spinal muscular atrophy (SMA) will be presented at the 2015 American Academy of Neurology 67thAnnual Meeting. The late-breaking abstract titled “SMN2 splicing modifier […]

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 8/1/2014

Isis Pharmaceuticals Initiates Phase 3 Study of ISIS-SMNRX in Infants with Spinal Muscular Atrophy

— ENDEAR Trial To Enroll 110 SMA Infants at Sites Globally — Dosage of First Infant Will Trigger $18 Million Milestone Payment  CARLSBAD, Calif., Aug. 1, 2014 – Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today the initiation of a pivotal Phase 3 study evaluating ISIS-SMNRx in infants with spinal muscular atrophy (SMA), the most common […]

Read more 3/10/2014

Trophos announces top-line results of pivotal trial of olesoxime in spinal muscular atrophy

Trophos announces top-line results of pivotal trial of olesoxime in spinal muscular atrophy Promising results for the maintenance of motor function in spinal muscular atrophy (SMA) – a rare, serious and debilitating pediatric-onset neurodegenerative disease Marseille, France, March 10, 2014 – Trophos today announces that top-line results from a pivotal clinical trial of its lead […]

Read more 2/21/2014

ISIS PHARMACEUTICALS REPORTS INTERIM RESULTS FROM ISIS-SMNRX MULTIPLE DOSE STUDY IN CHILDREN WITH SPINAL MUSCULAR ATROPHY

ISIS PHARMACEUTICALS REPORTS INTERIM RESULTS FROM ISIS-SMNRX MULTIPLE DOSE STUDY IN CHILDREN WITH SPINAL MUSCULAR ATROPHY Average increase of 3.7 points observed in muscle function score in SMA children treated with 9 mg of ISIS-SMNRx ISIS- SMNRx increases SMN protein in children with SMA On track to initiate Phase 3 study in children with SMA […]

Read more 1/22/2014

Joint Program in Spinal Muscular Atrophy Enters First Stage of Clinical Development

South Plainfield, NJ, January 22, 2014 – PTC Therapeutics, Inc. (NASDAQ: PTCT), the SMA Foundation, and Roche (SIX: RO, ROG; OTCQX: RHHBY), announced today that their joint research program in Spinal Muscular Atrophy (SMA) has entered the first stage of clinical development aiming to assess safety and tolerability of an oral compound in healthy volunteers. […]

Read more 11/18/2013

United States Senate Unanimously Passes the National Pediatric Research Network Act (NPRNA) Supporting SMA Research

United States Senate Unanimously Passes the National Pediatric Research Network Act (NPRNA) Supporting SMA Research Headed to the President’s desk today for signature, the legislation establishes a network of research consortia to focus on pediatric rare diseases, specifically including SMA.   Friday, November 15, 2013   Unanimously passed by the U.S. Senate on Thursday night, […]

Read more 9/19/2013

Isis Reports Follow-Up Data From ISIS-SMN Rx Phase 1 Study In Children With Spinal Muscular Atrophy

Isis Reports Follow-Up Data From ISIS-SMN Rx Phase 1 Study In Children With Spinal Muscular Atrophy Improvements in muscle function continue to be observed up to fourteen months after a single dose CARLSBAD, Calif., Sept. 19, 2013 /PRNewswire/ — Isis Pharmaceuticals, Inc.(NASDAQ: ISIS) announced today that follow-up preliminary data from a single dose, open-label Phase […]

Read more 4/23/2013

ISIS Pharmaceuticals Initiates a Clinical Study of ISIS-SMNRx in Infants with Spinal Muscular Atrophy

ISIS Pharmaceuticals Initiates a Clinical Study of ISIS-SMNRx in Infants with Spinal Muscular Atrophy  Isis to Earn $3.5 Million Milestone Payment   CARLSBAD, Calif., April 23, 2013 – Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced the initiation of a Phase 2 study of ISIS-SMNRx in infants with spinal muscular atrophy (SMA).   SMA is a severe and […]

Read more 3/21/2013

Data from Phase 1 Study of ISIS-SMNRx in Children with SMA Presented at American Academy of Neurology Meeting

Isis hosted an investor event and live webcast at 7:30 am PT on Thursday, March 21 in San Diego CARLSBAD, Calif., March 20, 2013 – Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced that data from the Phase 1 study of ISIS-SMNRx in children with spinal muscular atrophy (SMA) were presented today at the 65th American Academy […]

Read more 11/7/2012

Nature Medicine: “Call in the Backup” By Elie Dolgin

By Elie Dolgin The most common genetic killer of infants, a disease known as spinal muscular atrophy, is caused by mutations in a single gene. The human genome contains its own backup system—near-identical copies of the defective gene—yet these secondary sequences rarely get used correctly. As Elie Dolgin finds out, drug companies hope to change […]

Read more 11/1/2012

ISIS Pharmaceuticals Initiates Phase 1b/2a Study of ISIS-SMNRx in Patients with Spinal Muscular Atrophy

Ongoing Phase 1 Study Shows ISIS-SMNRx Well-Tolerated in Children with SMA CARLSBAD, Calif., November 1, 2012 – Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced the initiation of a Phase 1b/2a study evaluating ISIS-SMNRx in children with spinal muscular atrophy (SMA).  SMA is a severe and rare genetic neuromuscular disease characterized by muscle atrophy and weakness and […]

Read more 8/24/2012

Orphan Drugs Show Blockbuster Potential

The Economic Power of Orphan Drugs, (Thomson Reuters, August 23, 2012), reveals that today’s blockbuster drugs are not the only money makers by comparing the total projected value of orphan and non-orphan drugs from 1990 to 2030. This analysis is based on a peer-reviewed study from Drug Discovery Today. Following are some of the key […]

Read more 7/24/2012

SMA featured on the Charlie Rose Brain Series

The Charlie Rose Brain Series explores the study of the human brain. The most recent episode on motor neuron disorders features SMA. Included on the panel of scientists and researchers is Dr. Richard Finkel, Chief of Neurology at Nemours Children’s Hospital and a principal investigator for the SMA Foundation’s Pediatric Neuromuscular Clinical Research (PNCR) Network. […]

Read more 2/13/2012

Odd Couplings – Story by Rick Mullins, Chemical Engineering & News – Volume 90 Issue 7 | February 13, 2012

Drug Firms Engage in Nontraditional Research Partnerships in a Bid to Get Closer to the Patient In the pantheon of companies competing in the health care market, Proteus Biomedical is unique. A start-up based in Redwood City, Calif., Proteus has developed technology to attach a biodegradable digital device to a pill. After a patient swallows […]

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 2/3/2012

ProteinTech Interview with The Spinal Muscular Atrophy Foundation

Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disease characterized by motor neuron loss and muscle atrophy and weakness. The disease is caused by deletion or mutational inactivation of the SMN1 gene that encodes survival of motor neuron (SMN) protein, critical to motor neuron health and survival. The disease typically presents in early infancy and […]

Read more 1/4/2012

Biogen Idec and Isis Pharmaceuticals Announce Global Collaboration for Antisense Program Targeting Spinal Muscular Atrophy

Biogen Idec and Isis Pharmaceuticals Announce Global Collaboration for Antisense Program Targeting Spinal Muscular Atrophy — Biogen Idec has Option to Develop and Commercialize Promising Compound for Most Common Genetic Cause of Infant Mortality — — Biogen Idec’s Expertise in Neurology to Aid in Rapid Development of ISIS-SMNRx —  SMA is a genetic neuromuscular disease characterized […]

Read more 1/4/2012

Biogen May Spend as Much as $299 Million on Isis Treatment for Children

Biogen May Spend as Much as $299 Million on Isis Treatment for Children Bloomberg News by Robert Langreth January 4, 2012 Biogen Idec Inc. (BIIB) will gain Isis Pharmaceuticals Inc. (ISIS)’s drug for an inherited muscle-weakening disease that afflicts children, in a development deal that may cost as much as $299 million. Biogen, the world’s biggest maker […]

Read more 12/26/2011

Salvar a Arya

El País by Sandro Pozzi December 26, 2011 Las cifras ayudan a entender la batalla que libran Dinakar Singh y Loren Eng, millonario y exsocio del banco Goldman Sachs, y su esposa, Loren Eng, por su hija Arya. Entre los más de 312 millones de habitantes de EE UU, solo 9.000 tiene la enfermedad infantil […]

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 11/8/2011

A Thousand More

A family is determined to give their disabled son a whole and vital life. In the midst of a great burden, one small child – with a seemingly endless supply of love – is the blessing that holds a family together. See the project at http://mediastorm.com/training/a-thousand-more

Read more 9/7/2011

Bloomberg Markets: “To Save a Child”

Video – September 7, 2011 For the full story, please see the October edition of Bloomberg Markets.

Read more 6/22/2007

FSMA Announces Major Breakthrough in Development of Drug Candidate for Currently Untreatable Disease

June 22, 2007, Libertyville, IL – Families of Spinal Muscular Atrophy (FSMA) is pleased to announce the selection of a Clinical Candidate for Spinal Muscular Atrophy through its program being conducted at deCODE chemistry. At the same time FSMA is now extending its contract with deCODE to continue work towards an Investigational New Drug (IND) […]

Read more 3/29/2007

NIH/NINDS Director Highlights Remarkable Scientific Advances in SMA Research in Congressional Hearing

Washington, D.C. – March 29, 2007 – In Senate Subcommittee on Labor-HHS-Education Appropriations hearings this week, National Institute of Neurological Disorders and Stroke Director, Story C. Landis Ph.D., testified on the remarkable scientific advances that have been made in spinal muscular atrophy research over the past decade. This is an excerpt from her remarks: A decade […]

Read more 3/20/2007

Treatment Extends Survival in SMA Mouse – Researchers Optimistic About HDAC Inhibitors

story by Robinson, Richard Spinal muscular atrophy (SMA) — a juvenile form of motor neuron disease leading to weakness and wasting of voluntary muscles — is caused by a loss of the SMN1 gene, but even the most severely affected patients retain a functional copy of an almost identical gene, SMN2. In mice, increasing SMN2 […]

Read more 2/22/2007

Treatment Extends Survival in Mouse Model of Spinal Muscular Atrophy.

Drug therapy can extend survival and improve movement in a mouse model of spinal muscular atrophy (SMA), new research shows. The study, carried out at the NIH’s National Institute of Neurological Disorders and Stroke (NINDS), suggests that similar drugs might one day be useful for treating human SMA. “This study shows that treatment can be […]

Read more 11/10/2006

Spinal Muscular Atrophy Foundation Receives CNF Advocacy Award of Merit

New York, NY – November 10, 2006 – The Spinal Muscular Atrophy (SMA) Foundation announced today that it has received the 2006 Advocacy Award of Merit from the Child Neurology Foundation (CNF). The award was presented to the SMA Foundation during the Annual Meeting of the Child Neurology Society in Pittsburgh on October 20. Founded […]

Read more 10/11/2006

Lexicon Genetics Awarded Additional Grant for Study of SMA

The Woodlands, Texas – October 11, 2006 – Lexicon Genetics Incorporated (Nasdaq: LEXG) announced today that its research program to identify targets that may be important in the development of drugs to prevent or treat spinal muscular atrophy (SMA) has been extended for an additional year by the United States Army Medical Research & Materiel […]

Read more 6/7/2006

SMA Foundation Announces Collaboration with PTC Therapeutics

New York, NY – June 7, 2006 – The Spinal Muscular Atrophy Foundation today announced that they have initiated a research and development collaboration with PTC Therapeutics, Inc. (PTC). The collaboration is designed to leverage PTC’s proprietary Gene Expression Modulation by Smallmolecules (GEMS) technology to identify and develop new small molecule therapeutics for use in […]

Read more 5/20/2006

New Push for NIH Funding Urged

By Jonathan D. Rockoff Sun Reporter WASHINGTON, D.C.— May 20, 2006 – Senators urged research advocacy groups and universities yesterday to step up efforts to win more federal funding for the National Institutes of Health, the federal agency that funds most of the country’s medical research but is facing a third consecutive year of cuts. […]

Read more 12/26/2005

The Today Show, December 26, 2005, Featuring the SMA Foundation

On Monday, December 26, 2005, NBC’s The Today Show featured one family’s fight against thgenetic motor neuron disease Spinal Muscular Atrophy and the impact SMA has on parensearching for a treatment for their child. The show highlighted the daily struggle that 6-year-old Arya faces battling the effects of SMA and how she and her parents, […]

Read more 12/23/2005

New Motor Neuron Center Will Tackle Neurodegenerative Diseases

The birth of any organization dedicated to reducing human suffering is fueled by hope and propelled by the desire to achieve extraordinary goals; this was certainly true on Nov. 2 when Columbia’s Motor Neuron Center was launched. The gathering of about 40 leading researchers and clinicians from different disciplines at CUMC and Morningside, committed to […]

Read more 9/14/2005

Drug Provides Glimmer of Hope for Children Suffering Fatal Genetic Disease

Stanford University News by Krista Conger Ching Wang, MD, PhD, didn’t sign up for his pediatric neurology residency in 1990 to watch children die. But, as in nearly any medical specialty, there are some fatal diseases for which no effective treatment exists. Frustrated after delivering grim news to one too many sets of parents, Wang […]

Read more 9/1/2005

SMA Foundation Makes the Case for Research in Rare Disease

By Debbie Strickland The errant gene has been isolated, the National Institutes of Health has made the disease a priority and now a young foundation is pitching biotech companies to develop a cure for spinal muscular atrophy, a disabling, often fatal genetic disorder that afflicts some 25,000 Americans. Dinakar Singh and Loren Eng launched the […]

Read more 8/1/2005

SMA: Race Is On to Conquer Devastating Childhood Disease

SMA: Race is on to Conquer Devastating Childhood Disease When a young child is slow to meet developmental milestones such as rolling over, crawling, taking his first steps – or perhaps reaches them and then slows or slides back in development – parents grow concerned and look for explanations. For about 1 out of every […]

Read more 3/28/2005

Forbes: For Arya

-by Robert Langreth A Wall Street Power Couple Race to Find a Cure for Their Daughter Before It Is Too Late Arya Singh was born in March 2000, sat up and crawled on schedule but never really walked right. Pediatricians said at first that she was simply a late bloomer. But at 17 months she […]

Read more 3/7/2005

Deadly Disorder Kills Kids, But Lacks Funding

NEW YORK — March 7, 2005 – Two-year-old Owen Cain cannot lift his head or move his limbs. He’s fed through a tube, and even the simple act of sitting in a chair can trigger an alarming breathing spasm. The child has Spinal Muscular Atrophy, or SMA – the leading genetic killer of infants and […]

Read more 1/5/2004

Neurology Today: “Advocacy for Neurological Disease: The ‘Family Business’ No Family Wants”

By Gina Shaw They don’t have MD or PhD degrees. They don’t run research laboratories, direct hospitals, or oversee major academic medical centers. Yet they have helped changed the course of neurological research, driving tens of millions of research dollars – both private and federal – into the quest for treatments and cures for diseases […]

Read more 10/28/2003

The New York Times: “A Deadly Disease of Infants Attracts New Research Money,” by Anahad O’Connor

When Ellen Goldstein of Brooklyn gave birth last November to her only child, Owen, medical tests offered no clues that five months later he would be crippled by a deadly and irreversible genetic disease. Tests shortly before Owen was born revealed no abnormalities, and a physical evaluation right after his birth showed he was in […]

Read more 4/25/2003

North County Times: “Cure for Genetic Disease Requires Money,” by George Eng

Fifty years ago today, James Watson’s and Francis Crick’s scientific paper ignited a revolution in modern science. Fidgeting with cardboard cutouts and X-ray photographs, Watson and Crick discovered the spiral staircase structure of DNA. Their description of the double-helix sparked a torrent of research into DNA that today has us on the verge of finding […]

Read more 3/18/2003

Pittsburgh Post Gazette: “Funding Needed to Cure a Disease that Makes Children Waste Away”

By Loren A. Eng Fifty years ago, Francis Crick and James Watson ignited a revolution in modern science. Toying with a collection of cardboard cutouts and X-ray photographs, the two scientists discovered the structure of deoxyribonucleic acid, known as DNA. Their spontaneous observation of the “double helix” sparked a torrent of research into DNA that […]

Read more 3/4/2003

Parents Magazine: Parents Support Circle, “Arya, 3, Has Spinal Muscular Atrophy”

Read more 1/21/2003

Nightline: UpClose with Ted Koppel. “Arya Singh.”

ABC NEWS UPCLOSE: ARYA SINGH ANCHORED BY: TED KOPPEL WITH GUESTS: LOREN ENG AND DINAKAR SINGH JANUARY 21, 2003 TED KOPPEL, ABC NEWS (OC) “Up Close” tonight, a young girl, a two-year-old, whose name is Arya. She lives in New York City, with her parents and baby brother. Arya has a genetic disease that the […]

Read more 12/29/2002

The New York Times: “When Not Knowing a Celebrity Could Prove Fatal”, by Adam Cohen

By ADAM COHEN DECEMBER 29, 2002 When Loren Eng, a briskly efficient Stanford Business School graduate, lobbies Congress for research funds for the disease that is crippling her 2-year-old daughter, she takes along almost everything she needs to make an airtight case. She has figures showing that the disease, spinal muscular atrophy, is badly underfinanced […]

Read more 9/19/2002

WABC-TV, New York: “Family Helping Others Learn More About SMA.”

New York, WABC, September 19, 2002 — For parents, few things are more difficult than watching their child struggle with a serious illness. When a muscle disease threatened their daughter, a frustrated family looked for a treatment and then found a way to help others facing the same crisis. Now, ABC7’s On Call with Dr. […]

Read more 9/17/2002

Columbia University Press: “Columbia University & Columbia-Presbyterian Med Ctr Open New SMA Clinic”

WHEN & WHERE: Tuesday, September 17, Noon Harkness Pavilion 180 Fort Washington Avenue New York NY WHAT: The Departments of Neurology Columbia University and at Columbia Presbyterian Medical Center are celebrating the opening of the SMA (spinal muscular atrophy) Clinic, a center devoted to the treatment and management of a group of diseases that affect […]

Read more