The SMA drug pipeline has grown dramatically in just over a decade. Of the 17 programs in the pipeline, seven of these are in clinical trials, and two of those are in Phase 3 clinical trials. Reaching and achieving FDA approval is a major hurdle in our path to a treatment and cure. And, consistent […]
Update on SMA-FDA Interactions Read More »
The Family Friendly Research Poster Session is one of the most anticipated events of our Annual SMA Conference. Each year, Cure SMA invites researchers from the SMA Researcher Meeting, which is held in parallel with the family conference, to present their findings to families in way that is accessible to them. During the event, families
2015 Family Friendly Poster Session Presenters Announced Read More »
F. Hoffman-La Roche, PTC and the SMA Foundation recently provided an update on the clinical development of RG7800, an investigational SMN2 splicing modifier that is being studied as a potential treatment for spinal muscular atrophy (SMA): “In November 2014, Roche initiated the Moonfish trial (Phase 1b/2a) based on supportive results from a trial in healthy
Update on Clinical Development of RG7800 Read More »
Over the past few months, Cure SMA has announced a total of $1.85 million in new research funding. Our model is designed to fund both a breadth and a depth of projects, in order to continuing building up both a breadth and depth of programs in the drug pipeline. This includes funding for: Basic research,
Cure SMA Funds $1.85 Million in New Research Grants in 2014-2015 Read More »
Cure SMA, the leading organization dedicated to the treatment and cure of spinal muscular atrophy (SMA), recently provided an additional $445,000 grant toward a gene therapy for SMA at Nationwide Children’s Hospital. Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at Nationwide Children’s, and his team have made promising strides toward therapies
The US House of Representatives Energy and Commerce Committee has released a new 21st Century Cures discussion draft. This is a revised and expanded version of the first draft that was released in January. The 21st Century Cures initiative was created to investigate how the government can help “accelerate the discovery, development, and delivery of
New 21st Century Cures Discussion Draft Released Read More »
The deadline to register for our Annual SMA Conference is just two weeks away. Registration will close on Friday, May 9. Over 1,000 people have already registered—don’t miss your chance to be part of the action! The Annual SMA Conference is the largest SMA conference in the world for families, researchers, and medical providers. The
Registration for the 2015 Annual SMA Conference Closes in Two Weeks Read More »
Cure SMA has recently released an updated version of our latest care series booklet, The Musculoskeletal System. As with all our care series booklets, The Musculoskeletal System was written by medical professionals who specialize in SMA, and reviewed by families to ensure that it is clear and easy to understand, and that it addresses the
Cure SMA Publishes New Care Series Booklet on Musculoskeletal System Read More »
The Spring 2015 issue of Compass is now available online. As part of our most recent round of funding, we made five grants totaling $640,000 for basic research. Basic research investigates the cause and biology of SMA, in order to identify the most effective strategies for drug discovery. We also use basic research funding to
Spring 2015 Issue of Compass Now Available Read More »
The Annual SMA Conference will bring together well over a thousand families and researchers from around the world. During the conference, several sessions are set aside specifically for families and leading researchers to interact. One of the most important of these sessions is the Research Q&A Session that closes the conference. A dozen experts will
Annual SMA Conference to Feature Panel of Leading SMA Drug Experts Read More »
