Independent.ie on MSN14h
‘Every day counts’ – call to add screening for rare muscle-wasting condition to newborns’ ‘heel-prick’ testA majority of people have supported the urgent implementation of early screening for a genetic disease that causes severe ...
LancsLive on MSN16h
'We waited months for answers over our baby's condition - now he'll never be able to walk'Use precise geolocation data and actively scan device characteristics for identification. This is done to store and access ...
In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...
Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...
Use precise geolocation data and actively scan device characteristics for identification. This is done to store and access ...
Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and latent forms of myostatin in order to inhibit myostatin activation.
A Prescription Drug User Fee Act (PDUFA) target action date for apitegromab has been set for Sept. 22, 2025.
A rare genetic condition, Spinal Muscular Atrophy (SMA) affects one in 7,000 people in India. It mostly affects children and ...
Video: Spinal Muscular Atrophy: When is Surgical Intervention Appropriate? —How does one time surgery for scoliosis in children with pediatric spinal muscular atrophy get a maximum benefit ...
Chugai Pharma’s Evrysdi tablets 5mg receives Japanese approval to treat spinal muscular atrophy: Saturday, March 29, 2025, 11:00 Hrs [IST] Chugai Pharmaceutical Co., Ltd. announ ...
A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the ...
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