Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. 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 ...

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 ...

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 ...

- 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 ...

A 5-year-old British boy suffering from the rare disease spinal muscular atrophy (SMA) has miraculously started walking four years after receiving the world’s most expensive gene therapy. On the 31st, ...

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 ...

Please provide your email address to receive an email when new articles are posted on . At the time of last follow up, 82.6% of children born with SMA type 1 from 2016 on were still alive. Of the 140 ...

"Our preliminary findings show that German children with SMA, despite significant physical disability, have surprisingly good subjective HRQoL." – Erik Landfeldt, MSc, PhD Previous studies have found ...

Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended ...

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 ...