Neurology

The professor from National Taiwan University Hospital discussed his work in improving diagnoses in Pompe disease and AADC deficiency.

The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the importance of accessibility in clinical trials.

Review top news and interview highlights from the week ending March 18, 2022.

The draft guidance from the Center for Biologics Evaluation and Research outlines safety considerations throughout the clinical development program timeline.

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

Data presented at MDA 2022 show improvements in motor and pulmonary function and patient-reported outcomes.

Sarepta also presented updated data from Study 101 of SRP-9001 at MDA 2022.

The new designation follows an IND clearance and fast track review in February 2022.

Myrtelle’s gene therapy previously demonstrated initial efficacy in the disease in February 2022.

New data presented at MDA 2022 showcased positive motor and bulbar function data.

The global head of research and development at Atara Biotherapeutics discussed the future of MS research.

Review top news and interview highlights from the week ending March 11, 2022.

The therapy’s IND has been cleared and a first in-human trial is expected to begin later this year.

Patients demonstrated a median reduction of 63.5% in CSF NfL levels 6 months after treatment.

The senior vice president of clinical development at Passage Bio discussed biomarker activity seen in the IMAGINE-1 trial.

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

All patients in the first cohort exhibited increases in APOE2 CSF protein expression.

The chief scientific officer of the CMT Research Foundation discussed the foundation’s mission.

Review top news and interview highlights from the week ending March 4, 2022.

David Taylor, PhD, an assistant professor in the department of molecular biosciences at UT Austin, discussed the Cas9 research conducted between 2 labs at UT Austin.

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

The chief scientific officer of FibroBiologics discussed efficacy findings from a phase 1/2 trial.

The professor from National Taiwan University Hospital discussed the importance of Rare Disease Day for raising awareness.

Clinical and industry leaders share their perspectives on the importance of collaboration in developing treatments for rare diseases.

Crystal Proud, MD, outlines advances in technology and research in neuromuscular diseases.

Review top news and interview highlights from the week ending February 25, 2022.

The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the organization’s initiative to advance genetic testing.

Cory Nicholas, PhD, cofounder and chief executive officer, Neurona Therapeutics, discussed the company’s lead program in mesial temporal lobe epilepsy.

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

The chief scientific officer of FibroBiologics discussed the benefits of fibroblast cell technology in MS.