Ryan Flinn

Apitegromab, 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.

The 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.

SMA, 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.

Risdiplam 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.

According to a study published in JAMA Pediatrics, screening newborns for spinal muscular atrophy (SMA) and starting treatment immediately can significantly improve health outcomes compared to infants who are diagnosed and begin treatment months later.