While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be ...

A new clinical trial led in part by a Lurie Children’s Hospital doctor is showing promise for children with Dravet syndrome, ...

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...

Metagenomi Therapeutics, Inc. ( MGX) TD Cowen 46th Annual Health Care Conference March 2, 2026 11:10 AM EST ...

The drug was finally approved in September 2025 through the FDA’s accelerated approval pathway, based on improvement in knee extensor muscle strength, for adults and children with Barth syndrome who ...

Zahra Mahmoudjafari explains how cell and gene therapy programs succeed with strong infrastructure, pharmacy leadership, ...

"Beast Games" winner Jeff Allen hopes finding a cure for his son's rare disease will unlock new treatments for other brain conditions.

Please refer to the earnings press release issued this morning and our quarterly filing with the SEC for additional details. Revenue for the year ended 12/31/2025 was $16.1 million compared to $27.1 ...

Enrollment for liMeliGhT, the first and largest gene therapy registrational trial for broad retinitis pigmentosa (RP) patients, was completed, ...

The latest firm caught between the FDA’s shifting demands for “gold standard” science and regulatory flexibility for rare disease therapies, Uniqure NV saw its shares plummet after disclosing that U.S ...

Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary blindness, hemophilia and, recently, Huntington's disease, a fatal neurological ...

Human pluripotent stem-cells (HPSCs)—including human embryonic stem-cells and human induced pluripotent stem-cells—are reshaping the landscape of regenerative medicine.1,2 In Parkinson's disease, ...