SMA Patients on Apitegromab Still Showing Benefits at Three-Year Mark
August 19th 2024Apitegromab, an investigational drug for spinal muscular atrophy, demonstrated sustained patient benefits and maintained safety over a three-year period in a phase II study, with 88% of participants maintaining or improving motor milestones.
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Novel SMA Drug Delivery System Gets FDA Greenlight to Expand Trial Enrollment
August 14th 2024The system, called ThecaFlex DRx, allows subcutaneous administration of Spinraza (nusinersen) directly into the cerebrospinal fluid, and aims to replace the current administrative route through lumbar punctures.
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SMA Treatments Save Lives and Money, but Economic Barriers Hinder Access
July 19th 2024SMA, a rare genetic neurodegenerative disorder affecting approximately 1 in 10,000 newborns, is typically fatal for untreated children by age two. However, the approval of disease-modifying medications have transformed the treatment landscape.
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Risdiplam Five-Year Follow-Up: 91% of SMA Children Alive, Hitting Milestones
July 11th 2024Risdiplam is an oral small molecule treatment that impacts both the central nervous system and peripheral tissues. Patients receive the medicine daily in liquid form either by feeding tube or by mouth. The drug was first approved by the FDA in 2020 for patients as young as two-months old.
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