Dossier
Catholic faith
Coverage of Catholic faith in the Nexus archive.
- STAT+: The quest to save Grace — and clear the way for rare disease patients everywhere
Matt Wilsey developed a gene therapy to treat his daughter Grace's NGLY1 deficiency, an ultra-rare genetic disorder, but the treatment caused her to be hospitalized. Wilsey's efforts, which involved assembling scientists and advocates, aim to create a model for curing other rare diseases.