Neurology

Furthermore, the majority of patients treated with the gene therapy showed improved myelination as measured by T-2-weighted MRI.

Melissa Spencer, PhD, spoke about the emerging role of adeno-associated virus as a delivery system for gene therapies targeting muscular dystrophies, and the challenbges associated with them.

Sharon Hesterlee, PhD, the chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.

William Chou, MD, president and chief executive officer of Passage Bio, discussed feedback from a recent Type C meeting with the FDA.

Review top news and interview highlights from the week ending October 25, 2024.

Crystal Proud, MD, the director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, spoke about the enhancements to newborn screening for the disease.

The chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.

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

For the patients treated at the high dose, an average improvement of 18 points from baseline was observed in UPDRS Part 3 “off” medication score at 26 weeks posttreatment.

Review top news and interview highlights from the week ending October 18, 2024.

The phase 1/2 EPISOD1 study, which is taking place in the US, will provide participants with a short immunosuppression regimen before administration of the gene therapy.

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

Benitec reported that the first patient showed a 35% reduction, constituting clinically meaningful improvement, in total score on the Sydney Swallow Questionnaire.

The REVEAL adolescent and adult trial and the separate phase 1/2 REVEAL Pediatric Study are both evaluating the gene therapy TSHA-102 in Rett syndrome populations.

Review top news and interview highlights from the week ending October 11, 2024.

Paul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA, discussed the growing interest in gene therapy for the annual meeting.

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

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the growing interest in gene therapy for attendees of the annual meeting.

As cell therapy investigations mature in the field of AD, some programs show promising signs of efficacy.

Review top news and interview highlights from the week ending October 4, 2024.

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

Daniel Hart, PhD, the senior director and head of technology development at Epic Bio, discussed potential applications of the new technology.

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

CSF analyses from 7 patients treated in the study showed reductions of more than 80% in NAA levels from baseline.

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

The company pointed out that today, September 30, is LGMD Awareness Day.

John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, discussed several still-investigational drugs and therapies for DMD.

Review top news and interview highlights from the week ending September 27, 2024.

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

John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, discussed the complicated choice doctors, patients, and families now face with a wide range of treatment options available.