mld
The Philadelphia Family Helping to Drive Research on MLD
Catherine Offord | May 1, 2018 | 4 min read
Though Calliope Joy’s disease is too far progressed to be treatable, her parents have helped other children with metachromatic leukodystrophy get access to an experimental therapy.
Families of Children with Rare Diseases Fuel Gene Therapy Research
The Scientist | May 1, 2018 | 10+ min read
Crowdfunding can power investigations into diseases that would otherwise receive little attention.