Neurology

In a mid-cycle review meeting, the agency also noted that the review committee found no major deficiencies with the BLA package.

The clinical neurophysiologist at Children’s National and the professor of neurology at The Ohio State University Wexner Medical Center discussed the evolving SMA treatment landscape.

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

The company’s CEO announced via LinkedIn that the company is shutting down its operations, citing a “difficult funding environment.”

The chief scientific officer of the Parkinson’s Foundation discussed how broad genetic testing through PD GENEration is uncovering valuable prognostic and diagnostic insights in Parkinson disease.

The chief scientific officer of the Parkinson’s Foundation discussed the foundation’s efforts to integrate whole genome sequencing into PD GENEration and expand access across diverse global populations.

Linda Marbán, PhD, the CEO of Capricor Therapeutics, discussed future plans for the product after the promising data presented at MDA’s 2025 conference.

Review top news and interview highlights from the week ending May 2, 2025.

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

The CEO of Capricor Therapeutics discussed future plans for the product after the promising data presented at MDA’s 2025 conference.

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

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

Emerging gene and cell therapies aim to slow Parkinson disease progression. Explore clinical updates on seven promising candidates in development.

Matthew Alexander, PhD, a neuromuscular expert, discussed the evolving landscape of targeted therapies in muscular dystrophies, from fibrosis and glycosylation to combination strategies with gene therapy.

The chief scientific officer at Precision Biosciences discussed the durable potential of ARCUS gene editing and the company’s next steps toward in-human trials for Duchenne muscular dystrophy.

Linda Marbán, PhD, the CEO of Capricor Therapeutics, discussed clinical data presented at MDA’s 2025 meeting.

The chief scientific officer at Precision Biosciences discussed the ARCUS gene editing platform and its potential application in Duchenne muscular dystrophy through a durable, AAV-delivered therapeutic strategy.

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

The FDA’s decision was supported by data indicating overall survival benefit for patients with brain tumors from the phase 1 BrainChild-03 clinical trial.

The CEO of Capricor Therapeutics discussed clinical data presented at MDA’s 2025 meeting.

Notably, AMT-130 has previously been granted regenerative medicine advanced therapy, orphan drug, and fast track designations by the agency.

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

During the 12-month study, Descartes-08 was deemed well-tolerated.

Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio, discussed the genetic basis of oculopharyngeal muscular dystrophy, as well as the primary challenges in diagnosing and treating the condition.