Neurology

In observance of International Pompe Day, held annually on April 15, catch up on the past year’s news and expert insights related to gene therapies in development for this rare disease.

Review top news and interview highlights from the week ending April 14, 2023.

The professor for human genetics and neurology at the University of Miami Miller School of Medicine discussed past, current, and future approaches to diagnosing and treating neuromuscular diseases.

Matthew Gornet, MD, spine surgeon from The Orthopedic Center of St. Louis discussed data seen with IDCT cell therapy.

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

The professor for human genetics and neurology at the University of Miami Miller School of Medicine discussed the trend towards gene-targeted approaches in the neuromuscular disease space.

REGENXBIO’s RGX-202 previously received orphan drug and rare pediatric disease designations for DMD in late 2021 and early 2022, respectively.

The executive vice president and chief research officer of MDA discussed the barriers to FDA approval currently facing those developing treatments for ultra-rare diseases.

The director of the center for gene therapy at Nationwide Children's Hospital discussed the prospect of gene therapy approvals in Duchenne muscular dystrophy and the associated challenges that remain to be addressed.

Peter Marks, MD, PhD, director, Center for Biologics Evaluation and Research, FDA, discussed his keynote address at the 2023 MDA Conference. c

Review top news and interview highlights from the week ending April 7, 2023.

Study authors Matthew Charman, PhD, and Matthew D. Weitzman, PhD, elaborated on the findings and implications of their research.

The spine surgeon from The Orthopedic Center of St. Louis discussed recent data on IDCT from the phase 1/2 DGX-A01 study.

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

The professor and director of the Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center at University of Washington School of Medicine discussed the PDC group and collaborating with advocacy groups.

No treatment-emergent adverse events experienced were deemed related to AstroRx, although 9 serious TEAEs did occur.

The neurology resident at Penn Medicine spoke about the preparations taken in the lead up to tofersen’s PDUFA date, which is set for April 25, 2023.

The co-CEO of BrainStorm Cell Therapeutics spoke about the current standards of care in ALS and the potential of NurOwn to address unmet needs.

Review top news and interview highlights from the week ending March 31, 2023.

The associate professor of neurology at Columbia University spoke about the importance of identifying patients with ALS-associated gene mutations as early as possible.

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota spoke about preclinical research with an iPS cell-derived product.

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

The associate professor of neurology at Columbia University spoke about the current and future treatment landscape in ALS.

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota spoke about the session she chaired at the 2023 MDA Clinical and Scientific Conference.

In November 2022, BrainStorm received a refusal to file letter from the FDA for the biologics license application of NurOwn.

The co-CEO of BrainStorm Cell Therapeutics spoke on the supporting data for the investigational ALS cell therapy that will be discussed at the meeting, and expectations for it.

The company's persistence in bringing NurOwn to the market amidst negative regulatory feedback has garnered an AdComm meeting.

The nurse practitioner from Washington University in St. Louis discussed the practical considerations in gene therapy track she chaired at the 2023 MDA Conference.

The child neurologist and neuromuscular physician at Virginia Commonwealth University also discussed the session she chaired at the 2023 MDA Clinical and Scientific Conference.

Review top news and interview highlights from the week ending March 24, 2023.