We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Topline results from a phase 3 clinical trial showed an ...

SMA type 2, previously known as Dubowitz disease, is a hereditary disease that affects motor neurons. It typically becomes noticeable around the ages of 6 to 18 months, when children with the ...

Evrysdi, also known as risdiplam, is an oral medicine used to treat spinal muscular atrophy (SMA) in children and adults. SMA is a genetic disease that is passed down through families. It leads to ...

The recent availability of 3 FDA-approved treatments for patients with spinal muscular atrophy (SMA) has dramatically altered patient outcomes and provided a wealth of new avenues for SMA research. A ...

In a multidisciplinary follow-up study, researchers analyzed bone health decline and the effects of nusinersen in patients with spinal muscular atrophy (SMA) types 2 and 3. SMA is a progressive, ...

Researchers evaluated oxygen saturation and arterial CO2 variables to screen for sleep disordered breathing in children with SMA type 1-3. Of the measures evaluated, only an oxygen desaturation index ...

- Majority (74%) of patients with non-ambulatory Type 2 and Type 3 SMA achieved a clinical improvement in Hammersmith Functional Motor Scale Expanded (HFMSE) after 12 months - New exploratory analysis ...

Scholar Rock Holding Corporation (NASDAQ:SRRK) President & CEO Jay Backstrom presented at the J.P. Morgan Healthcare Conference. The discussions mainly focused on apitegromab for spinal muscular ...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new 5-year data confirming the sustained efficacy and safety profile ...

More than 30 types of muscular dystrophy have been identified. They all cause problems with movement due to muscle protein defects caused by genetic mutations. Muscular dystrophy is a group of ...