Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease
Condition(s):Infantile-onset Pompe DiseaseLast Updated:November 1, 2023Recruiting
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Condition(s):Infantile-onset Pompe DiseaseLast Updated:November 1, 2023Recruiting
Condition(s):X-linked RetinoschisisLast Updated:June 12, 2023Completed
Condition(s):Idiopathic Parkinson’s DiseaseLast Updated:November 10, 2022Completed
Condition(s):Pompe Disease Infantile-OnsetLast Updated:June 6, 2023Recruiting
Condition(s):Alpha-1 Antitrypsin DeficiencyLast Updated:March 28, 2019Completed
Condition(s):Hemophilia ALast Updated:December 15, 2023Recruiting
Condition(s):Leber Congenital AmaurosisLast Updated:December 28, 2017Completed
Condition(s):Retinitis PigmentosaLast Updated:May 3, 2023Recruiting
Condition(s):Hemophilia ALast Updated:May 15, 2023Active, not recruiting
Condition(s):Leber’s Hereditary Optic NeuropathyLast Updated:December 8, 2023Active, not recruiting
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