Spinal Muscular Atrophy treatment is evolving fast, with gene therapy, oral drugs, and next-gen muscle enhancers offering new hope and long-term outcomes. With each breakthrough in SMA treatment, we ...

RegenXBio will fill its coffers with up to $250 million by monetizing more royalty rights for Novartis-partnered Zolgensma as well as anticipated payments linked to a gene therapy nearing approval ...

A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the treatment of older patients with spinal muscular atrophy (SMA).

Understanding a drug’s mechanism of action (MOA) can greatly increase the likelihood of successful clinical trials by identifying biomarkers of patient response and providing a rationale for ...

Signal strength of reports of onasemnogene abeparvovec (Zolgensma) at the System Organ Class (SOC ... on onasemnogene abeparvovec has predominantly concentrated on its mechanism of action, clinical ...

“We are encouraged by the results of this AD trial exploring how targeting the disease from different angles with different mechanisms of action impacts disease markers,” said Kreesten ...

A research team now describes a new mechanism of action that kills cancer cells through ferroptosis. In a first, a Bochum-based team has produced a substance capable of sending cancer cells into ...

Zolgensma (onasemnogene abeparvovec-xioi) is a brand-name drug prescribed for spinal muscular atrophy in certain children. Zolgensma comes as a liquid suspension for intravenous (IV) infusion. It ...

Currently in clinical development are pipeline assets with novel mechanisms of action that are suggested to improve muscle ... and/or have a history of treatment with Zolgensma. Biohaven notes that ...

Zolgensma (onasemnogene abeparvovec-xioi) is a prescription gene therapy drug that’s used to treat spinal muscular atrophy in certain children. A healthcare professional will administer the drug ...

Novartis presented new data that continue to support the clinical benefits of Zolgensma (onasemnogene abeparvovec), the only one-time gene therapy for the treatment of spinal muscular atrophy (SMA).