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SMA Press


About SMA: SMA Press

FDA Approves label Extension for Evrysdi for Infants with SMA Under 2 Months Old

May 31, 2022

The SMA Foundation’s partners, Roche and PTC Therapeutics, announced today that the FDA has approved a label extension for Evrysdi(risdiplam) to include babies under two months old with SMA. The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that pre-symptomatic babies treated with Evrysdi for at least one…

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Scholar Rock shares community update on SAPPHIRE, a clinical trial evaluating apitegromab in non-ambulatory patients with Type 2 and 3 SMA

November 30, 2021

Today Scholar Rock shared a community update on SAPPHIRE, a clinical trial evaluating apitegromab in non-ambulatory patients with Type 2 and 3 SMA: Dear SMA Community, We are excited to announce the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of apitegromab in those living with Spinal Muscular…

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

January 4, 2012

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…

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Salvar a Arya

December 26, 2011

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…

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

December 19, 2011

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…

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

November 29, 2011

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…

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A Thousand More

November 8, 2011

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

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Bloomberg Markets: “To Save a Child”

September 7, 2011

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

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

June 22, 2007

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)…

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

March 29, 2007

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…

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

March 20, 2007

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…

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Treatment Extends Survival in Mouse Model of Spinal Muscular Atrophy.

February 22, 2007

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…

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