New developments in treatments for spinal muscular atrophy (SMA) over the last few years have changed what is possible for people with SMA, allowing them to achieve new developmental milestones. However, these treatment ...
During our past fiscal year—from July 1, 2019 to June 30, 2020—Cure SMA funded more than $9 million in new and ongoing research and care initiatives. This money will be used strategically to ...
Recently, Cure SMA and colleagues at Novartis Gene Therapies published a manuscript titled, “Economic burden of spinal muscular atrophy: an analysis of claims data” in the Journal of Market Access & Health Policy. ...
Dear Members of the SMA Community, As we celebrate National Family Caregivers Month this November, Biogen acknowledges the vital role caregivers have in our society, including within our spinal muscular atrophy (SMA) community. ...
Each year since 2017, Cure SMA has conducted a Community Update Survey. This online questionnaire is sent to all individuals with spinal muscular atrophy (SMA) and caregivers of children with SMA in the ...
Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced 6-month interim results from their clinical program for its ...
Through Cure SMA’s partnership with NeurologyLive, a new patient-centered article, “Early Intervention in SMA: How Access to Therapy Will Alter Disease Management As We Know It,” is now available for viewing ...
Cure SMA has published a paper, titled “Spinal Muscular Atrophy and COVID-19: Guidance and Resources for Providers,” in partnership with NeurologyLive. The article highlights findings from a recent survey conducted by ...
Novartis Gene Therapies today announced new interim data from the ongoing Phase 3 STR1VE-EU clinical trial for Zolgensma® (onasemnogene abeparvovec). The data demonstrates that patients with spinal muscular atrophy (SMA) Type ...
Dear Members of the SMA Community, We continue to work with the healthcare community to help people and families living with SMA get the support they need. As such, we wanted to share ...
Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the U.S. Food and Drug Administration (FDA) has ...
Dear SMA Patient Advocacy Community, As part of our ongoing partnership and following your request to receive updates about the risdiplam clinical development program, we are delighted to share with you a much-anticipated ...
