Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

Nearly all children with spinal muscular atrophy who started on Evrysdi before symptom onset are walking after three years, ...

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

Novartis said new interim data from an ongoing phase clinical trial for its $2.1 million-per-patient gene therapy Zolgensma showed spinal muscular atrophy patients experienced significant therapeutic ...

Biogen also reported new real-world evidence and progress on the development of a novel device to enhance the patient treatment experience CAMBRIDGE, Mass., June 30, 2023 (GLOBE NEWSWIRE) -- Biogen ...

Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, discusses how physicians position different therapies for patients with ...

As their 8-month-old daughter prepares for gene therapy treatment, a Coon Rapids family is bracing for three months of ...

Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, highlights the current FDA-approved therapies for spinal muscular atrophy.

The Rare Disease Therapeutics Market is experiencing an unprecedented wave of innovation, driven by gene therapies, RNA-based ...