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Transformative therapies for rare diseases with high unmet needs.
Innovative partnerships that create new opportunities in the rare disease field.
On our commitment to the patients, families, and communities we serve.
Complete DiGeorge Anomaly
Children with complete DiGeorge Anomaly are born without a thymus gland, resulting in primary immunodeficiency. The disease is almost uniformly fatal within the first 24 months of life if untreated.
Farber disease is an ultra-rare lysosomal storage disease caused by a mutation in both alleles of the ASAH1 gene, resulting in the deficiency of the lysosomal enzyme acid ceramidase.
April 3, 2019
Enzyvant today announced that its investigational regenerative treatment (RVT-802) in late-stage development for congenital athymia will be featured during a platform presentation, at a symposium, and in a poster session at the Clinical Immunology Society (CIS) 2019.
March 21, 2019
The FDA has granted Rare Pediatric Disease and Fast Track designations for RVT-801, an investigational enzyme replacement therapy.
February 21, 2019
Rachelle is a proven leader and operator with deep experience bringing valued medicines to market in complex, fast-moving business environments,