Pompe Disease Infantile-Onset Clinical Trials
3 recruiting trials for Pompe Disease Infantile-Onset. Eligibility criteria explained in plain English.
3
Total Trials
3
Recruiting Now
0
Phase 3 Trials
2
Sponsors
Recruiting Trials
RECRUITINGNCT05619900
Registry of Patients Diagnosed With Lysosomal Storage Diseases
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the...
Sponsor: University of California, San FranciscoEnrolling: 2501 location
RECRUITINGPhase 1NCT04532047
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in...
Sponsor: University of California, San FranciscoEnrolling: 101 location
RECRUITINGNCT06833489
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Since 2017, more than 250 analyses performed at the Molecular Genetics Laboratory of the Timone Enfant Hospital have yielded negative results in patients with rare genetic muscle...
Sponsor: Assistance Publique Hopitaux De MarseilleEnrolling: 501 location