Spinal Muscular Atrophy (SMA) causes progressive muscle weakness and, without treatment, can limit life expectancy to just ...

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 ...

Patients treated with OAV101 IT showed an increase from baseline in HFMSE total score compared with sham control. Topline data were announced from a phase 3 trial evaluating intrathecal onasemnogene ...

Spinal muscular atrophy (SMA) can result in muscle weakness, breathing and swallowing issues and a loss of mobility in ...

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 ...

PHILADELPHIA--(BUSINESS WIRE)--Exegenesis Bio, a rapidly growing global genetic medicines company, is pleased to announce the presentation of clinical efficacy and safety data from its EXG001-307 ...

The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.

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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 ...

This Easter season, fundraising and other efforts are being made to help a 9-year-old girl and her family get a wheelchair ...

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 ...