The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…
With the addition of clinics in Canada and Israel, the Angelman Syndrome Foundation (ASF) now has an international network of Angelman Syndrome Clinics staffed with Angelman syndrome (AS) experts. The Children’s Hospital of Eastern Ontario (CHEO) in Ottawa, Canada, and the Edmond and Lily Safra…
If successful, an extensive pilot screening study afoot in Australia for Angelman syndrome and three other rare genetic disorders could lead to large-scale testing. With a contribution from the Victorian Medical Research Acceleration Fund, the Angelman Syndrome Foundation (ASF) and the Foundation for Prader-Willi Research (FPWR)…
The Angelman Syndrome Clinic has opened at Rush University Children’s Hospital in Chicago, Rush University Medical Center and the Angelman Syndrome Foundation (ASF) announced in a news release. It is the first such clinic in Illinois, bringing to eight the number in the United States. The…
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