Topic: muscular dystrophy
Synchron started a clinical trial of its brain-machine interface that will gauge how the technology restores communication in people who are paralyzed.
A CRISPR-based gene activation technique has prevented and reversed muscular dystrophy symptoms in mice.
Targeting DUX4 might improve the success of immunotherapies, scientists at Fred Hutchinson Cancer Research Center suggest.
Fulcrum plans to reposition the p38 mitogen-activated protein kinase inhibitor as a treatment for facioscapulohumeral muscular dystrophy.
A new Atlas-incubated startup focused on rare forms of muscle diseases has got off a $50 million series A with experienced executives at the helm.
Another gene therapy alliance shows that Sarepta won’t allow an issue with its lead gene therapy candidate to dampen its enthusiasm for the field.
The gene therapy suppresses expression of a mutated protein and encourages production of a functional version, thereby lessening muscular problems.
Sarepta licenses five new gene therapies to its muscular dystrophy portfolio from Myonexus—and takes an option on buying the company.
Sarepta is hoping to avoid the data-quality controversy that arose over its Duchenne muscular dystrophy drug Exondys51 last year with its next-generation drug golodirsen.
A University of Missouri-led team developed a new gene transfer method to treat Duchenne muscular dystrophy.