Glycogen storage disease due to acid maltase deficiency, late-onset clinical trials in California
3 active Glycogen storage disease due to acid maltase deficiency, late-onset trials with a site in California. Describe your situation to see which ones look like a fit.
- A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
- A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
- Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Guides for people searching for a trial
- How to search for a trial without losing your mindWhere to look, what to gather first, and how to read what you find.
- Why people get turned down — and what to do nextWhat eligibility criteria are for, what keeps people out, and where to go when the first door closes.
- How your biomarkers decide which trials you qualify forEGFR, HER2, BRAF, PD-L1 and the rest — what they are and why they increasingly gate access.
- What Phase 1, 2 and 3 actually mean for youWhat each phase is testing, and how to weigh an early-phase trial against a later one.
- 10 questions to ask before you say yesTreatment, time, cost, safety and your rights. Worth bringing to a screening visit.
- You matched with a trial. What happens now?Phone screens, the screening visit, informed consent, and day one.
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