Clinical trials: ADA SCID
Severe Combined Immunodeficiency Due to ADA Deficiency
Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)
STRIMVELIS is a medicinal product that restores adenosine deaminase (ADA) function in hematopoietic cell lineages, thereby preventing impaired immune function.
- Trial name
- NCT03232203
- Testing
- STRIMVELIS
- Where
- Italy
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Fondazione Telethon
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International