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SMA Press
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 MoreWebcast 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 MoreDear 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 MoreWASHINGTON, 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 MoreResults 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— 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 MoreTrophos 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 MoreISIS 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 MoreSouth 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 MoreUnited 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 MoreIsis 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…
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