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The U.S. Food and Drug Administration recently approved Xenpozyme (Olipudase alfa) for intravenous infusion in pediatric and adult patients with Acid Sphingomyelinase Deficiency (ASMD), a rare genetic disease that causes …
FDA Approves Treatment for Rare Genetic Disease Read More
WASHINGTON, D.C. — The U.S. Food and Drug Administration recently approved Voxzogo (vosoritide) injection to improve growth in children five years of age and older with achondroplasia and open epiphyses …
FDA Approves First Drug to Improve Growth in Children with Most Common Form of Dwarfism Read More
PHILADELPHIA, PA — Amicus Therapeutics (Nasdaq: FOLD) announced that the data from the Phase 3 PROPEL pivotal trial, assessing the efficacy, safety and tolerability of AT-GAA in adults with late-onset …
The Lancet Neurology Publishes Pivotal Phase 3 PROPEL Study Results of AT-GAA in Late-Onset Pompe Disease Read More WASHINGTON, D.C. — The U.S. Food and Drug Administration recently approved Besremi (ropeginterferon alfa-2b-njft) injection to treat adults with polycythemia vera, a blood disease that causes the overproduction of red …
FDA Approves Treatment for Rare Blood Disease Read More WASHINGTON, D.C. — The U.S. Food and Drug Administration announced it recently awarded 11 new clinical trial research grants, equaling more than $25 million of funding over the next four …
11 Grants Awarded to Clinical Trials to Develop New Medical Products for Rare Disease Treatments Read More
EXTON, PA — Castle Creek Biosciences, Inc. recently announced a research collaboration with Mayo Clinic to advance discovery and pre-clinical development of investigational gene therapy candidates for the treatment of …
Castle Creek Biosciences Expands Gene Therapy Platform for Rare Genetic Connective Tissue Disorders Read More
WASHINGTON, D.C. — The U.S. Food and Drug Administration recently announced the approval of Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients 1 year of age and older with …
FDA Approves New Treatment for Pompe Disease Read More