The muscles waste away because of a gene defect. Eventually, the muscle cells die and are replaced with fatty and fibrous tissue that cannot generate any strength. Duchenne and Becker muscular dystrophies often are diagnosed in childhood, but other types may not appear until middle age or later. Duchenne muscular dystrophy is the most common childhood dystrophy; it occurs almost exclusively in boys and usually is diagnosed between the ages of two and six. Muscular dystrophy is considered incurable, and some types, like Duchenne, significantly shorten the life span of the patient.
CTRF's 50 billion cell investigational clinical trials include Duchenne,
Becker and limb
girdle muscular dystrophies.
Is MTT a 'treatment' for muscular dystrophy?
No. A treatment is defined as an FDA-approved procedure. CTRF is in Phase II investigational clinical trials involving a relatively small number of closely monitored subjects over a 21 month period (three months pre-transplant and 18 post-transplant). Data � which is randomized and double-blinded � is collected and, as each trial concludes, promptly submitted to the FDA. The Foundation will also publish this data in a peer review journal so that academics can review
Are there any other treatments on the horizon for muscular dystrophy?
Unfortunately not. Researchers are conducting a wide variety of experiments
� many of them on animals and, in particular, in mice. Even if these
experiments suddenly showed promise it would likely take at least five to 10
years to receive FDA permission to make it available as a treatment. Current
medical care for muscular dystrophy includes physical therapy, palliative
surgical procedures and steroids, despite the fact that the anabolic steroids
prednisone and deflazocort have serious, long-term side effects and can lead to
an earlier death. Neither prednisone nor deflazocort has been approved by the
FDA to treat muscular dystrophy-afflicted individuals.
Why do people volunteer for CTRF's investigational clinical
trials?
The decision to volunteer for CTRF's clinical trials is a deeply personal one. Each individual undoubtedly has a unique set of reasons. All have at least this much in common:
The most acute form of muscular dystrophy is Duchenne. Parents enroll their DMD children in CTRF's clinical trials for several reasons, among which are: