Roche has officially launched two Phase 2 clinical trials testing RO7034067 (also known as RG7916). The trials will begin recruitment in October. Earlier this month, Roche first announced plans to begin these Phase 2 trials in response to favorable results from a Phase 1 study in healthy volunteers. RO7034067 is an orally available drug that […]
Roche Launches New Phase 2 Clinical Trials Read More »
Over the last decade, we’ve seen many advances in spinal muscular atrophy research, from new techniques in gene therapy to drugs that show promise in slowing or stopping the progress of the disease. With such great promise in the research landscape, we know that no single group can develop a treatment for SMA alone—it requires
Catch Up on Research News from the 2016 Family Friendly Research Poster Session Read More »
Roche, PTC Therapeutics, and the SMA Foundation have released a community update on the development of their SMN2 splicing modifier clinical development program: Dear SMA community, Roche, together with our collaboration partners PTC Therapeutics and the SMA Foundation, would like to provide you with an update on our SMN2 splicing modifier clinical development program. Please
Roche Provides New Update on Clinical Development of RG7916 Read More »
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 350 attendees. The goal of the meeting is to create open communication of early, unpublished scientific data, accelerating the pace of research. The meeting also furthers research by building productive collaborations—including cross-disciplinary
2016 SMA Researcher Meeting Summary: Regulation of SMN Protein Expression and Function Read More »
Recently, Cure SMA confirmed three new board members and one new committee member. These new members will provide additional leadership in the areas of advocacy, FDA/regulation, and research. We extend a warm welcome to new board members Kelly Cole; Doug Kerr, MD, PhD, MBA; and Thomas Murray, PhD. We also extend a warm welcome to
Cure SMA Welcomes Aboard New Board and Committee Members Read More »
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 350 attendees. The goal of the meeting is to create open communication of early, unpublished scientific data, accelerating the pace of research. The meeting also furthers research by building productive collaborations—including cross-disciplinary
2016 SMA Researcher Meeting Summary: SMA Therapy Development Read More »
The Summer 2016 issue of Compass is now available online. As part of our most recent round of funding, we’ve announced $704,000 in funding for four new drug discovery grants. Individuals with SMA don’t produce survival motor neuron (SMN) protein at high enough levels due to a mutation in the survival motor neuron 1 (SMN1)
Summer 2016 Issue of Compass Now Available Read More »
We’re thrilled to announce a new partnership that will bring Cure SMA’s resources in front of The Mighty‘s wide-reaching readership. We will now have a growing home page on The Mighty and appear on many stories on the site, allowing us to get many more people involved with our organization. The Mighty is a story-based
We’re Partnering With The Mighty! Read More »
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 350 attendees. The goal of the meeting is to create open communication of early, unpublished scientific data, accelerating the pace of research. The meeting also furthers research by building productive collaborations—including cross-disciplinary
The SMA Researcher Meeting is the largest research meeting in the world specifically focused on SMA. This year we had a record setting 350 attendees. The goal of the meeting is to create open communication of early, unpublished scientific data, accelerating the pace of research. The meeting also furthers research by building productive collaborations—including cross-disciplinary
