Megan’s affliction, Pompe disease, is exceedingly rare. The course of the disease varies by patient, but it causes children’s muscles to weaken and, eventually, their lungs may fail.
We are looking for individuals with a diagnosis of Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) to take part in a research study which will tell us whether late-onset Pompe disease or ...
Sanofi has added another string to its Pompe disease therapy bow ... the market four years later to treat late-onset Pompe in children aged over eight. In 2010, Lumizyme's label was expanded ...