Neurology

Astellas Pauses Gene Therapy Trial for X-Linked Myotubular Myopathy

Following a voluntary pause, a patient experiencing a liver AE has died and the trial is now on clinical hold.

Developing MCO-010 for Retinal Degenerative Diseases

Samarendra Mohanty, PhD, cofounder and president, Nanoscope Therapeutics, discussed the company’s technological platforms and RESTORE trial data.

New Gene Therapy Collaboration Targets Huntington Disease

NeuExcell and Spark Therapeutics each add to their gene therapy pipelines with the collaborative program.