Spinraza is a medication used in children (including newborns) and adults to treat spinal muscular atrophy. Treatment begins with four loading doses, followed by one maintenance dose every 4 months.
Oligonucleotide (oligo) therapies have been in development for decades. Recent advances in molecular design, chemistry, ...
Dubai’s Medcare Hospital becomes world’s first to treat adult Spinal Muscular Atrophy (SMA) patient outside the US ...
Cenrifki prevents MS disease progression and Itvisma delivers a healthy gene-to-cell nuclei, addressing the root cause of SMA.
A retrospective, single-center study out of the Czech Republic tested the safety and efficacy of unilateral interlaminar fenestration (UILF) on the convex side in cases of SMA that also exhibit severe ...
The findings, say the researchers, confirm that repeated intrathecal injections of the gene-targeting therapy are safe, adding to the limited data on the long-term effect and safety of the treatment ...
Biogen has partnered with Alcyone Therapeutics to improve the delivery of its spinal muscular atrophy drug Spinraza, handing over $10 million upfront for a global license to an implantable device.
Ractigen Therapeutics, a clinical-stage biopharmaceutical company focused on developing next-generation RNA-based medicines, today announce ...
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