BioSpace10h
Roche’s Evrysdi Becomes First Pill Cleared for Spinal Muscular Atrophy
The FDA on Wednesday approved the industry’s first-ever tablet treatment for spinal muscular atrophy— Roche ’s SMN2 splicing ...
The American Journal of Managed Care1d
Risdiplam Tablet for Spinal Muscular Atrophy Receives FDA Approval
The 5 mg risdiplam tablet provides the same efficacy and safety for spinal muscular atrophy as the currently available oral ...
The Independent1d
Massillon community unites to gather Valentine's Day cards for 7-year-old Vinny DeMando
An effort is ongoing to collect valentines for Massillon resident Vinny DeMando, 7, who has muscular dystrophy. Cards are ...
Genetic mechanisms underlying Duchenne muscular dystrophy point to potential treatments
Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...
Managed Healthcare Executive1d
Sarepta Announces Positive Results for Elevidys, Gene Therapy for Duchenne
Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...
FiercePharma1d
Roche scores FDA nod for Evrysdi tablets, bolstering convenience edge over rivals
Over the past nine years, patients with spinal muscular atrophy have seen the number of FDA-approved treatment options for ...
Urine test offers simpler diagnosis for mitochondrial disorders, study finds
New research at the University of Alberta may make it easier to diagnose serious diseases that disrupt the body's energy ...
Analysts Predict Up to ~450% Surge for These 2 ‘Strong Buy’ Penny Stocks
Some of the market’s biggest winners start off as the smallest players. While trillion-dollar tech giants like the ...