Advertisement

Topics

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

16:57 EST 8 Nov 2017 | News-Medical.net

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.

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

NEXT ARTICLE

More From BioPortfolio on "New gene therapy trial for X-linked myotubular myopathy offers hope to children"

Advertisement
Quick Search
Advertisement
Advertisement

 

Relevant Topics

Pediatrics
Pediatrics is the general medicine of childhood. Because of the developmental processes (psychological and physical) of childhood, the involvement of parents, and the social management of conditions at home and at school, pediatrics is a specialty. With ...

Clincial Trials
In a clinical trial or interventional study, participants receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices; procedures; or change...

Gene Therapy
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene th...