Neurology

Recent long-term data from the phase 2 extension has also demonstrated CAP-1002's efficacy in Duchenne.

Review top news and interview highlights from the week ending July 22, 2022.

Upstaza has been granted marketing authorization by the European Commission.

The principal investigator of the HOPE-2 trial discussed results of the trial in the non-ambulatory population of patients with Duchenne muscular dystrophy.

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

The professor of neurosurgery at Rush University Medical School discussed data seen in the phase 1/2 clinical trial of LCTOPC1.

Ultragenyx also exercised its option to acquire GeneTx, its partner in developing GTX-102.

Rajesh Pahwa, MD, FANA, FAAN, discussed balancing hope with current challenges with stem cell approaches in PD.

Review top news and interview highlights from the week ending July 15, 2022.

Patient recruitment for the clinical trial is currently underway at epilepsy centers throughout the US.

There were not enough data at 6 months to predict expanded disability status scale (EDSS) improvements after treatment.

The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed mutations the Parkinson’s Foundation has identified.

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

Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed the foundation’s mission.

Myrtelle’s rAAV-Olig001-ASPA was granted fast track, rare pediatric disease, and orphan drug designations in March 2022.

Two patients reached significantly higher cognitive development ages than seen in natural history 24 months after dosing.

Patients demonstrated a 7-point improvement above pre-treatment baselines on the North Star Ambulatory Assessment at 4 years post-treatment.

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

The global head of research and development at Atara Biotherapeutics discussed ATA188 ahead of anticipated data readouts this month.

Review top news and interview highlights from the week ending July 1, 2022.

A patient showed more than a 50% reduction in N-acetylaspartate (NAA) in brain white matter at 3 months post-treatment.

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

It will be the first AMN clinical study to utilize wearable technology for monitoring patient activity and outcomes.

Benjamin Harrison, BSc, PhD, assistant professor of biochemistry and nutrition at UNE, shared his thoughts on the research.

Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed the panel discussion she participated in at BIO 2022.