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2 articles from the last 30 days matching "trial results"

Clinical trialCLINICALTRIALSApr 14

Trial Results Posted: Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR) (NCT04046224)

Researchers tested a new gene therapy called ST-920 for Fabry disease in 36 patients. This treatment uses a modified virus to deliver instructions that help the body make an enzyme called alpha-galactosidase A, which people with Fabry disease don't produce enough of. The trial is now complete and tested whether different doses were safe and well-tolerated.

WHY IT MATTERSThis completed Phase 1/2 trial is the first human test of ST-920, meaning results could help determine if gene therapy can provide long-term relief for Fabry disease patients who currently require lifelong enzyme replacement infusions.
💬 Ask your doctorFabry diseaseRead →
PolicyRSSApr 13

FDA Reminds More Than 2,200 Sponsors and Researchers to Disclose Trial Results

The FDA sent reminders to over 2,200 companies and research groups telling them they must share their clinical trial results on a public website called ClinicalTrials.gov. This is a requirement, not optional. The FDA is making sure that information about how medical treatments work—whether they help patients or cause problems—gets shared publicly so everyone can see the results.

WHY IT MATTERSWhen companies hide negative trial results, patients may not know the full picture about whether a treatment actually works, which could affect decisions about whether to participate in a trial or use a medication.
ℹ Good to knowRead →

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