More attention should be given to evaluating the gastrointestinal symptoms of individuals with late-onset Pompe disease (LOPD) because they…
Articles by Forest Ray PhD
The type of genetic mutation carried by a Pompe disease patient appears to predict whether the disease is infantile-…
Spark Therapeutics has dosed the first patient in the Phase 1/2 RESOLUTE clinical trial of SPK-3006, an experimental gene…
The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography…
Amicus Therapeutics remains on track to announce top-line data from PROPEL — a Phase 3 trial of…
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the…
Combining exercise training with a high-protein diet improves exercise tolerance, pulmonary function, and quality of life among late-onset Pompe…
Treatment with Myozyme (alglucosidase alfa, marketed as Lumizyme in the U.S.) improves walking ability and may stabilize respiratory function in adults…
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure…