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 ...
Genentech, a member of the Roche Group, today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for Evrysdi (risdiplam) to treat adults and children 2 months and older with spinal ...
Genentech, a member of the Roche Group, shared two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 years with Type 2 or 3 spinal muscular ...
Biogen, Inc., today announced new results from NURTURE, the longest study of pre-symptomatic patients with spinal muscular atrophy (SMA), which is transforming expectations of early treatment with SPINRAZA (nusinersen). In ...
In the midst of the ongoing Coronavirus (COVID-19) global pandemic, Cure SMA is excited to announce its new funding for the Pediatric Neuromuscular Clinical Research Network (PNCRN). In 2020, Cure SMA will provide ...
New SPINRAZA® (nusinersen) Data Reinforce Sustained Efficacy and Longer-Term Safety Across Broad Range of SMA Patients Biogen Inc. recently announced additional data from the SPINRAZA (nusinersen) clinical development program that further demonstrate the ...
