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Consensus Guidelines Aim to Optimize DMD Gene Therapy Delivery
19 Apr 2025 02:18 GMT
… approvals in DMD and spinal muscular atrophy providing options with the potential …
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Gene therapy improves motor function in spinal muscular atrophy at 52 weeks
01 Apr 2025 22:46 GMT
… to 18 years with spinal muscular atrophy, data show.
The findings were … abeparvovec for patients with spinal muscular atrophy: Phase 3 randomized, sham-controlled …
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Chugai Pharma's Evrysdi tablets 5mg receives Japanese approval to treat spinal muscular atrophy
29 Mar 2025 06:09 GMT
… new formulation of the spinal muscular atrophy (SMA) treatment Evrysdi [generic name … States in February 2025.
Spinal muscular atrophy (SMA) is a genetic neuromuscular …
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HC allows Natco Pharma to make Swiss firm Roche’s spinal muscular atrophy drug in public interest
26 Mar 2025 14:18 GMT
… , an oral medicine for spinal muscular atrophy.
This effectively permits Natco Pharma … of the rare disease.
Spinal muscular atrophy is a neuromuscular disorder that …
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How Delhi High Court's latest verdict is a boon for spinal muscular atrophy patients
26 Mar 2025 07:55 GMT
… a breakthrough for Spinal Muscular Atrophy (SMA) patients, the … treatment for years.
Spinal Muscular Atrophy (SMA) is a rare … those suffering from spinal muscular atrophy, welcomed the decision saying … .
Aarav suffers from spinal muscular atrophy (SMA) Type 2, …
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FDA Grants Priority Review BLA for Spinal Muscular Atrophy Drug, Apitegromab
26 Mar 2025 01:27 GMT
… apitegromab, a treatment for spinal muscular atrophy (SMA), according to a news … serious adverse events recorded.
Spinal muscular atrophy is a rare genetic disease …
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Natco Pharma Allowed to Make Roche's Spinal Muscular Atrophy Drug in Public Interest
25 Mar 2025 13:11 GMT
… , an oral medicine for Spinal Muscular Atrophy (SMA).
In what could be …
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FDA Grants Priority Review for Biologics License Application (BLA) and EMA Accepts Marketing Authorisation Application (MAA) for Apitegromab as a Treatment for Spinal Muscular Atrophy
25 Mar 2025 11:43 GMT
… for people living with spinal muscular atrophy (SMA) who are receiving an … -targeted treatment candidate in spinal muscular atrophy (SMA) to demonstrate clinical success …
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6 Therapies on the Horizon for Spinal Muscular Atrophy
24 Mar 2025 05:41 GMT
… changed how clinicians treat spinal muscular atrophy. In 2016, Biogen’s Spinraza …
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Novartis Shares Positive Phase III Data for Spinal Muscular Atrophy Therapy
20 Mar 2025 21:33 GMT
… <18 years with spinal muscular atrophy (SMA).1
In the Phase …
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