Clinical Trials for Rare Diseases

Cell-selective, adeno-associated virus gene therapy DB-OTO restored hearing to normal levels within 24 weeks in a child born with profound genetic deafness due to variants of the otoferlin gene.

In recognition of International Women's Month, we're featuring this recent talk between Associate Editor Miranda Schmalfuhs and Marie Teil, Global Head of UCB’s Women of Childbearing Age Program. They speak about the specific challenges women with chronic illnesses face when accessing appropriate treatment and participating in clinical trials, UCB's Women of Childbearing Age Program and it’s most successful strategies, and much more.

Phase I/IIa HORNBILL trial results show Boehringer Ingelheim’s novel humanized monoclonal anti-Sema3A antibody was well tolerated and showed early signs of efficacy.

In recognition of Women's Health Month, we're featuring this recent talk between Associate Editor Miranda Schmalfuhs and uMotif's Chief Product Officer, Julia Lakeland, discuss new technologies improving patient engagement and reducing the emotional and logistical burdens of participation, ethical considerations that should be addressed when implementing those technologies, while ensuring patient privacy, and much more.

Systematic review of over 300 randomized clinical trials sought to determine how often these values were overestimated in contemporary cardiovascular trials.

Johnson & Johnson’s novel targeted therapy TAR-200 produced a greater than 80% complete response rate in patients with Bacillus Calmette-Guérin–unresponsive, high-risk non–muscle-invasive bladder cancer with carcinoma in situ.