SMA Press
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 MoreLetter 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 MorePhase 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 MoreOrally 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 MoreIsis 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 MoreTrophos 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…
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