AveXis, Inc., a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today provided an update on interim data from the ongoing Phase 1 trial of AVXS-101 in spinal muscular atrophy (SMA) Type 1 as of September 15, 2016. The data were presented by Jerry Mendell, M.D., director […]
New data from the clinical program for nusinersen, an investigational treatment for spinal muscular atrophy (SMA), were presented by Biogen and Ionis Pharmaceuticals in the late-breaking session at the 2016 World Muscle Society Congress in Granada, Spain. The presentations included safety results from the interim analysis of the Phase 3 ENDEAR study in infantile-onset SMA
Biogen Presents New Data at World Muscle Society Congress Read More »
Twice this year, Cure SMA has had the opportunity to testify before the federal Advisory Committee on Heritable Disorders on the need for newborn screening for SMA. This committee, part of the Health Resources and Services Administration (HRSA), an agency of the U.S. Department of Health and Human Services, determines which conditions will be added
Cure SMA Announces Newborn Screening Initiative in Partnership with MDA Read More »
Biogen and Ionis Pharmaceuticals have provided the following community statement regarding the the completion of their NDA filing for nusinersen. Dear members of the SMA community, Today we have achieved a crucial step in the pathway to approval of nusinersen. We have completed the submission of our New Drug Application (NDA) to the Food and
Biogen and Ionis Release Community Statement on NDA Filing Completion Read More »
Biogen and Ionis today announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Biogen has also applied for Priority Review which, if granted, would shorten the review period of
Biogen Completes Rolling Submission of New Drug Application to FDA Read More »
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 »
