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Divided into 3 sections, this presentation provides insight into the complexities of developing and manufacturing biologics and discusses considerations for the development of biosimilars.
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STEER Data Open Door to SMA Gene Therapy for Wider Age Range of Children
March 19th 2025Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular atrophy (SMA) aged 2 to 17 years, who had previously been shut out of receiving gene therapy.
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Bustling Gene Therapy Pipeline for Neuromuscular Diseases Brings Thorny Questions to the Clinic
March 18th 2025The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but also logistical hurdles and a need for complex discussions between clinicians and families.
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