A note from Cure SMA: We’re passing on information about this new interview study to the families in our community. It provides an opportunity to make your opinions known to an influential group of decision makers, in keeping with our mission of supporting advocacy and awareness. Your participation in this is voluntary. In addition, Cure […]
Adelphi Values’ RO7350A Interview Study Read More »
Cure SMA has awarded a $50,000 drug discovery grant to Barrington Burnett, PhD, at Uniformed Services University of the Health Sciences. The award is for the project, “Slowing SMN degradation to treat SMA.” Individuals with SMA don’t produce survival motor neuron (SMN) protein at high enough levels due to a mutation in the survival motor
At the Annual SMA Conference, representatives from the six programs currently in clinical trials gave an update on their programs. Of these six programs, four— AVXS-101 (gene therapy), nusinersen, RG7800/RG7916, and LMI070—treat the underlying genetics of SMA. Gene therapy aims to replace the mutated in the SMN1 gene that causes SMA, and the other three
Cure SMA Releases 2016 Updates from Drug Programs in Clinical Trials Read More »
The ultimate goal in drug development is to get treatments approved for as many people as possible, as quickly as possible. Because these issues are complex, we want our community to be knowledgeable about the burden of proof required to support an FDA-approved treatment, and how that approval process works. As we get closer to
Cure SMA Posts Keynote Presentations on the NDA Process Read More »
Over the past few months, Cure SMA has announced a total of just over $2 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:
Cure SMA Funds $2 Million in New Research in 2015-2016 Read More »
The 2016 Annual SMA Conference, now in its 28th year, is set to kick off on June 16 and continue into a weekend full of programs and events that bring families, researchers, and healthcare providers together. This year’s conference is on track to have 25% more attendees than the last time we were at Disneyland!
The 2016 Annual SMA Conference is Here! Read More »
Cure SMA has awarded a $304,000 drug discovery grant to Livio Pellizzoni, PhD, at Columbia University. The award is for the project, “Pharmacological Inhibition of p38αMAPK as a Candidate Therapeutic Approach for SMA.” About the Project Dr. Pellizzoni and his colleagues at Northwestern University have identified a novel cellular pathway, called p38αMAPK, that is altered
For the eighth consecutive year, Cure SMA has received a grant from the National Institutes of Health (NIH) for the SMA Researcher Meeting. This year’s grant will go toward conference expenses for graduate students and fellows, who are younger researchers in training. With the help of this NIH grant, Cure SMA generously subsidizes the cost
Cure SMA Receives 2016 NIH Grant Read More »
Cure SMA is pleased to announce that Biogen will be the Presenting Sponsor for our 2016 Annual SMA Conference. This year’s conference is set to be a significant event for the SMA community, with over 1,400 families and researchers coming together in Disneyland, June 16-19. “Without the generosity and support of Biogen, it would not
Cure SMA Announces Biogen as Presenting Sponsor for the 2016 Annual SMA Conference Read More »
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 18 active programs. 14 pharmaceutical partners. 6 programs in clinical trials. 28 programs in the cumulative pipeline total, including 10 failures to date. An ever-increasing breadth of potential treatment approaches to SMA. Effective Treatments for All Ages, Stages and Types of
Cure SMA Releases June 2016 Update to the SMA Drug Pipeline Read More »
