FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration challenges, with key selection factors like patient age and gene copy ...

A retrospective, exploratory analysis identifies biomarkers predictive of spinal muscular atrophy (SMA) response to nusinersen and suggests the efficacy of using machine learning algorithms to ...

Evrysdi can stabilize motor function, improve swallowing ability, ease fatigue, and improve life quality for adults with SMA, ...

MISSISSAUGA, ON, April 15, 2021 /CNW/ - Hoffmann-La Roche Limited (Roche Canada) today announced that Health Canada has granted EVRYSDI Ⓡ (risdiplam) market authorization for the treatment of spinal ...

A positive effect of nusinersen treatment on motor function in ambulant pediatric and adult spinal muscular atrophy (SMA) patients during a 38-month period has been demonstrated by an analysis of data ...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today presented positive results from the primary analysis of the ongoing RAINBOWFISH ...

A study tested 38 children with SMA who took part in an eight-week breathing exercise program, with some also engaging their ...

Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, ...

First and only muscle-targeted SMA therapy to demonstrate statistically significant, clinically meaningful improvements on the gold-standard HFMSE scale (p=0.019) versus placebo, with consistent ...

A Paediatric neurologist shares insight into the condition after former Little Mix singer Jesy Nelson revealed her twins have ...

Scholar Rock Holding ((SRRK)) announced an update on their ongoing clinical study. Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst ...