Panelists discuss how combination therapies targeting different aspects of spinal muscular atrophy (SMA), such as myostatin inhibitors (targeting muscle) alongside SMN protein-enhancing treatments, ...
Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers ...
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, ...
After up to 4.8 years of continuous treatment with SPINRAZA, 100 percent of children treated pre-symptomatically were alive, and none require permanent ventilation Patients continued to maintain and ...
Spinal Muscular Atrophy, known as SMA Type 1, explained: A Paediatric neurologist shares insight into the condition after former Little Mix singer Jesy Nelson revealed her twins have been diagnosed ...
Editor’s note: This is an automatically generated transcript. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription. So it can be challenging to determine which ...
A single dose of a new formulation of the gene replacement therapy intrathecal onasemnogene abeparvovec ( OAV101 IT) improved movement ability in teens and children older than 2 years with spinal ...
The European Commission has given approval to a high-dose regimen of Spinraza (nusinersen) for people with 5q spinal muscular ...
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Rohit Sharma grabbed by woman in desperate attempt to save her daughter suffering from SMA type 2: What is the medical condition
A startling moment went viral recently when Indian cricket star Rohit Sharma was grabbed by a woman outside a team hotel. The ...
More than two years after the Government committed to adding SMA to newborn testing, families are still waiting ...
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