Neurology

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

In observance of Spinal Muscular Atrophy Awareness Month, held annually in August, we took a look back at the past year's news in gene and cell therapy for SMA.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

Review top news and interview highlights from the week ending August 1, 2025.

The opinion recommends against conditional marketing authorization for patients who are ambulatory.

The FDA also stated that it has concluded its investigation of the death an 8-year-old boy who was treated with Elevidys in Brazil.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The patient, an 8-year-old boy, was treated with Elevidys in Brazil.

Review top news and interview highlights from the week ending July 25, 2025.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed the ways ongoing research into next-generation DMD therapies aims to improve then.

The agency also put several of the company’s clinical trials on hold.

The company stated that it has informed the FDA about its decision, which will go into effect at close of business on July 22, 2025.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed benefits and eligibility for newer DMD treatment options for older patients.

Review top news and interview highlights from the week ending July 18, 2025.

The CGTLive® team highlights 5 therapeutics that are nearing major decisions by the FDA.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

According to Ultragenyx, the CRL relates to a need for additional CMC information and improvements and observations from inspections of manufacturing facilities.

Review top news and interview highlights from the week ending July 11, 2025.

In the CRL, the FDA stated that the statutory requirement for “substantial evidence of effectiveness” was not met by the BLA and that more clinical data will be needed.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how comprehensive care for DMD patients involves early diagnosis, steroid treatment, consideration of mutation-specific therapies, and more.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how gene therapy shows promise for DMD treatment.

SNUG01 is intended to deliver a copy of the human TRIM72 gene to neurons via an rAAV9 vector.

Review top news and interview highlights from the week ending July 4, 2025.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

Review top news and interview highlights from the week ending June 27, 2025.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.