Muscular Dystrophy Drug

June 22, 2014

Monique Griffen is no stranger to the doctor’s office. She suffers from muscle weakness that was progressively getting worse and doctors didn’t know why.

Monique Griffen/Suffers from Pompe disease: “Inflammatory myopathy was the one they thought I had for the ten years and at one point they thought I had lupus.”

But, recently she was correctly diagnosed with late-onset Pompe disease, a form of muscular dystrophy.

Monique Griffen/Suffers from Pompe disease: “It was a relief to know what it was because until you know what it is, you can’t really fight it.”

Monique’s fight began when she learned there are no cures for her disease. She enrolled in a clinical trial at the University of Florida where they were testing a new treatment for her condition. UF researchers monitoring Monique were encouraged with the results of the treatment.

Dr. Barry Byrne/UF Researcher: “She can do some activities of daily living that she was not able to do before.”

Now Lumizyme, the drug researchers used in treating Monique in the clinical trial, is approved by the FDA for all patients who suffer from late on-set Pompe disease. UF researchers are happy there is finally a treatment for people who suffer from this rare disease.

Dr. Barry Byrne/UF Researcher: “To develop a real specific treatment that actually shows some benefit is actually very rewarding because the patients are eternally grateful and that’s the most beneficial thing to all the people who are involved in the clinical care.”

Monique will need an infusion of Lumizyme every two weeks for the rest of her life, but she says it is well worth it. She is excited for other people with Pompe disease that will have access to the new drug.

Monique Griffen/Suffers from Pompe disease: “A lot of patients have been out there waiting for treatment can now get it, so it is really exiting for the whole Pompe community.”

Now Monique hopes to avoid all those doctor visits and hopefully continue to improve with each treatment.