Author Archives: Marisa Wexler MS

Equity Concerns as Medicaid for Spinraza, Zolgensma Varies by State

Within the U.S., there is marked state-to-state variation in guidelines for whether Medicaid will cover Spinraza (nusinersen) or Zolgensma (onasemnogene abeparvovec) treatments, according to a new study. This finding raises concerns about equitable access to these lifesaving therapies for spinal muscular atrophy (SMA), according to researchers. The…

Biogen Acquires New Experimental SMA Therapy BIIB115 From Ionis

Biogen has exercised an option to acquire BIIB115, an investigational treatment for spinal muscular atrophy (SMA), from Ionis Pharmaceuticals. SMA is a rare genetic condition caused by mutations that lead to abnormally low levels of the survival motor neuron protein, known as SMN. BIIB115, previously called ION306,…

Treatment Delays May Be Less Important Than Family Support

The COVID-19 pandemic led to delays in treatment with Spinraza (nusinersen) for many children with spinal muscular atrophy (SMA). However, these delays seem to have a lower impact on children’s functional skills when compared with family support, according to a small study in Italy. The study, “…

Weak Trunk Muscles, Spine Problems Need Early Attention in SMA

Children with spinal muscular atrophy (SMA) who have spine deformities or weak trunk muscles are more likely than others to have substantial limits to functional abilities and life activities, a study in patients ages 6 months to 15 years reported. Strengthening trunk muscles through regular exercise and physical therapy…

Novartis Wraps Zolgensma Price, Availability Negotiations in Canada

Novartis has completed negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) regarding pricing and availability of Zolgensma (onasemnogene abeparvovec), its approved gene therapy for spinal muscular atrophy (SMA), for pediatric patients. With this negotiation completed, individual public drug plans in Canada will now make final decisions about the coverage…