Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will…
Articles by Patricia Inácio, PhD
The U.S. Food and Drug Administration (FDA) has extended by three months its review of Sanofi Genzyme‘s application seeking approval…
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a…
In an effort to raise public awareness and increase understanding of Pompe disease, the 8th Annual International Pompe Day…
Amicus Therapeutics is seeking U.S. approval of AT-GAA, its investigational therapy for late-onset Pompe disease. The company has begun…
A Phase 1/2 clinical trial is recruiting adults with late-onset Pompe disease to test Audentes Therapeutics‘ investigational gene…
Two novel mutations in the GAA gene were linked with cerebral stroke in two siblings with late-onset…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However,…
Aging and vascular risk factors, not enzyme replacement therapy or the disease itself, may increase the risk of late-onset Pompe…