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About SMA: SMA Press
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…
Read MoreToday 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…
Read MoreISIS 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 MoreIsis 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 MoreBy 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 MoreOngoing 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 MoreThe 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 MoreThe 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 MoreDrug 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 MoreOn 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 MoreSpinal 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 MoreBiogen 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…
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