Pompe Disease Infantile-onset
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CLINICAL_TRIALS other · 2026-04-08Clinical Trial Update: Registry of Patients Diagnosed With Lysosomal Storage Diseases
This ClinicalTrials.gov registry entry describes ongoing recruitment for a registry of patients diagnosed with various lysosomal storage dis…
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CLINICAL_TRIALS trial_result · 2025-09-10Clinical Trial Update: Expanded Access for ATB200/AT2221 for the Treatment of IOPD
Expanded Access for ATB200/AT2221 to treat infantile-onset Pompe disease is available, per the sponsor Amicus Therapeutics.