Novartis to Open, at FDA Request, New Trial of Zolgensma for Older Patients
Novartis will open a new clinical trial investigating Zolgensma, given via an injection directly into the spinal canal,…
Articles by Grace Frank
FAQs About Evrysdi (Risdiplam)
Evrysdi (risdiplam) is an oral liquid treatment approved in the U.S. for infants through adults with spinal muscular atrophy…
Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say
While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands,…
FDA Asked for Newest Risdiplam Data to Support Broad Label, Roche Says
The U.S. Food and Drug Administration (FDA) asked for the SUNFISH clinical trial’s top-line data that necessitated a delay in…
FDA Issues No Fines for Data Problems in Zolgensma’s Application
The U.S. Food and Drug Administration has decided not to fine or otherwise sanction Novartis for manipulated data included in…
18 States in US Screening Newborns for SMA as Efforts Continue for More, MDA Says
Eighteen states are now screening newborns for spinal muscular atrophy (SMA), the most recent neuromuscular disease added to…
MDA Executive Discusses Gene Therapies, SMN2 Modifiers, and Challenges in Treating SMA
Gene therapies — after a tragedy kicked them back to the lab some two decades ago — are beginning to…
From Care Centers to Research Awards and Summer Camp, MDA Leads in Helping Others
With about $18 million expected to be awarded in 2020 for grants supporting research across neuromuscular diseases, the…
Parents of 2 Babies Treated with Zolgensma in July: We Made the Right Choice
The parents of two babies treated in July with Zolgensma, the first gene therapy approved for spinal muscular…
Risdiplam, Potential Oral Therapy for All SMA Types, Granted Priority FDA Review
The U.S. Food and Drug Administration (FDA) accepted Genentech‘s application requesting approval for risdiplam, its potential oral treatment…