spinal muscular atrophy

[Biohaven Pharmaceutical Holding Company Ltd.] Biohaven Pharmaceutical Holding Company Ltd. announced that it entered into a license agreement with Bristol Myers Squibb for the development and commercialization rights to taldefgrobep alfa, a novel, Phase III-ready anti-myostatin adnectin.

[Gene Therapy] The authors performed a systematic review of relevant publications and extracted median survival and details of experimental design. A random effects meta-analysis was used to estimate and compare efficacy.

[Cell Reports Medicine] The authors discuss the three currently licensed therapies for spinal muscular atrophy, briefly highlighting their respective advantages and disadvantages, before considering alternative approaches to increasing survival motor neuron protein levels.

[Gene Therapy] Researchers described Qatari experience with onasemnogene abeparvovec by reviewing the clinical outcomes of nine spinal muscular atrophy children.

[Stem Cell Reviews and Reports] The authors outline the current progress towards fully PSC-derived in vitro co-culture models of the human neuromuscular junction and discuss future directions and challenges that must be overcome in order to create reproducible fully PSC-derived models that can be used for developmental studies, disease modeling, and drug testing.

[Journal of Cachexia Sarcopenia and Muscle] Researchers showed that survival motor neuron (SMN)-deficient myoblasts readily fused with wild-type myoblasts, demonstrating fusion competency. Conditioned media from wild type differentiating myoblasts did not rescue the fusion deficit of SMN-deficient cells, suggesting that compromised fusion may have primarily been a result of altered membrane dynamics at the cell surface.