Isis Pharmaceuticals, a Ribonucleic acid-targeted (RNA-targeted) drug discovery and development company, recently updated the results from an ongoing open-label Phase 2 trial of ISIS-SMNRx in infants with a diagnosis of spinal muscular atrophy (SMA) type I. The company had previously reported results from this trial which included assessments of muscle function, event-free…
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Cure SMA, a non-profit organization focused on supporting research projects to find a cure for spinal muscular atrophy (SMA), has released an update on its drug pipeline of products currently under development to treat SMA. The organization has revealed its enthusiasm with both the increased number of projects…
Biogen, a Cambridge, Massachusetts based developer, manufacturer and marketer of innovative therapies for patients living with serious neurological, autoimmune and hematologic disorders, in collaboration with Isis Pharmaceuticals — a leader in discovery and and development of drugs based on antisense and RNAi technology and focused on RNA…
Over 600 walkers and runners helped raised more than $100,000 during the 15th Annual New England Chapter Walk-n-Roll, on Saturday, May 16, at the DCR Wompatuck Park in Hingham, MA. The participants in the event walked through 1.5 miles to support patients who suffer from spinal muscular atrophy (SMA), gathering money to fund research to…
The non-profit organization Cure SMA has been awarded a National Institutes of Health (NIH) grant to support the preparation of its SMA Researcher Meeting. This is the seventh consecutive year that the NIH has financed the conference, and the funds will be used to support registration and travelling expenses of young fellow…
F. Hoffman-La Roche in Switzerland, PTC Therapeutics and the SMA Foundation recently provided a critical update on RG7800 safety, an investigational oral therapeutic drug currently being evaluated for the treatment of spinal muscular atrophy (SMA). SMA is a rare, devastating motor neuron disease and one of the leading…
A 21st Century Cures discussion draft has been released by the U.S. House of Representatives Energy and Commerce Committee as a basis for discussing new methods of accelerating discovery, development and delivery of novel therapies and cures. The initiative was established to investigate different ways the government can support these advancements. The newly revised and expanded…
Nonprofit organization Cure SMA has awarded a $445,000 grant to fund research on a gene therapy for the treatment of spinal muscular atrophy (SMA) conducted at Nationwide Children’s Hospital. The additional funding was granted to the principal investigator of the Center for Gene Therapy, Brian Kaspar, PhD, as announced by the organization in a recent press release.
Dallas, TX based AveXis has announced that company co-founder John Carbona has resigned from his position as CEO. Mr. Carbona will remain a Director of AveXis. This announcement stems from a succession plan, the development of which has been in process as AveXis advances clinical development of its…
Previously thought to be a disease only of motor neurons, spinal muscular atrophy (SMA) is now also known to be influenced by support cells of the central nervous system. Specifically, astrocytes, which regulate synapse formation between neurons and provide metabolic support for neurons, are essential to the pathogenesis of SMA…
