Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...

To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...

Development strategies designed to reduce the unknowns so that cell and gene therapy innovators can develop robust processes ...

In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...

Gene mutations in acute myeloid leukemia (AML) cells can guide treatment options, and machine learning can rapidly guess the ...

In this interview, News Medical speaks with Olivier Negre, Chief Scientific Officer at Smart Immune, about how immunotherapy is being revolutionized.

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

A patient has died following treatment with Elevidys, the Sarepta Therapeutics product that is the only FDA-approved gene therapy for Duchenne muscular dystrophy, the company disclosed Tuesday.