Favorable interim results reported in stem-cell therapy trial

January 8, 2018 | Categories: Research | Tagged: , , ,

A therapy developed at UW shows promise in its first test in young boys who have an inherited deadly muscle disease. Favorable interim results have been announced in a small clinical trial of a stem-cell therapy to treat patients with a rare, deadly disease called myotubular myopathy. Read More

Gene replacement therapy that works in dogs to be tested in children

May 18, 2017 | Categories: Research | Tagged: , , , ,

SEATTLE – KIRO 7 has learned of a revolutionary treatment, here in Seattle, that has saved the lives of dogs and is just months away from being tested in children. In video obtained by KIRO 7, dogs that were destined to die show no signs of the disease after a single infusion of gene-replacement therapy.  The […] Read More

UW, family rewriting neuromuscular research

November 23, 2016 | Categories: Announcements | Tagged: , ,

Physicians and researchers at the UW Medicine Institute for Stem Cell and Regenerative Medicine have partnered up with a nonprofit foundation in one of the leading efforts to find a cure or treatment for a rare neuromuscular disease called myotubular myopathy (MTM). The inherited disease is almost exclusively found in males and becomes evident at birth. […] Read More

Childers displays a dog family tree showing those affected and unaffected by an inherited muscle disorder similar to X-linked myotubular myopathy in people.

Gene therapy leads to robust improvements in animal model of fatal muscle disease

January 22, 2014 | Categories: Research | Tagged: , ,

January 22, 2014 Preclinical studies show that gene therapy can improve muscle strength in small- and large-animal models of a fatal congenital childhood disease know as X-linked myotubular myopathy. The findings, appearing  as the cover story in the January 22, 2014 issue of Science Translational Medicine, also demonstrate the feasibility of future clinical trials of gene therapy for […] Read More