X Linked Myotubular Myopathy News and Research

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New gene-delivering workhorse could make gene therapy safer, more effective for muscle diseases

Genetic muscle diseases lead to progressive muscle wasting and often early death, with few treatment options and no cure.

9 Sep 2021

New editorial published following adverse events of gene therapy clinical trial

The Editor-in-Chief of Human Gene Therapy, the first journal devoted to the field of gene therapy, and one of the world's leading experts on gene therapy have co-authored a new editorial, Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy, in response to the news of two deaths in a now-halted gene therapy clinical trial.

3 Jul 2020

New gene therapy trial for X-linked myotubular myopathy offers hope to children

Ann & Robert H. Lurie Children's Hospital of Chicago is one of the few centers participating in ASPIRO, an international Phase 1/2 clinical trial of a gene therapy product called AT132 for X-linked myotubular myopathy – a rare disease characterized by severe muscle weakness, breathing difficulty and early death.

8 Nov 2017

Audentes Therapeutics closes $30 million Series A financing

Audentes Therapeutics, Inc., a recently founded biotechnology company dedicated to the development of innovative treatments for rare muscle diseases, today announced the closing of a $30 million Series A financing.

18 Jul 2013

New targeted enzyme replacement therapy can treat X-linked myotubular myopathy

A collaborative research team including a Medical College of Wisconsin (MCW) pediatric neuropathologist successfully mitigated some of the effects of a muscular disease by using a new targeted enzyme replacement therapy strategy from 4s3 Bioscience.

23 Jan 2013