About SMA:
SMA Press
About SMA: SMA Press
Scholar Rock announced that ISEMBYLD™ (apitegromab-mstn) was approved by the U.S. Food and Drug Administration (FDA) for the treatment of Spinal Muscular Atrophy (SMA) in adults and children 2 years of age and older who are currently receiving an SMN2-targeted treatment. ISEMBYLD showed a clinically meaningful improvement in motor function in the Phase 3 SAPPHIRE…
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…
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…
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…
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…
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…
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…
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
Video – September 7, 2011 For the full story, please see the October edition of Bloomberg Markets.
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)…
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…
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…