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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.
February 28, 2018
Re:GenerationHope raises awareness and funds for the complete DiGeorge Anomaly
January 8-10, 2018
Coinciding with the annual J.P. Morgan Healthcare Conference