New pill shows promise for rare genetic brain disorders
NCT ID NCT05758922
First seen May 25, 2026 · Last updated May 25, 2026
Summary
This study tested an experimental oral drug, AZ-3102, in 13 people aged 12 and older with GM2 gangliosidosis (Tay-Sachs or Sandhoff disease) or Niemann-Pick type C disease. The main goals were to check the drug's safety and how it moves through the body over 12 weeks. Participants were randomly assigned to receive the drug or a placebo, and those who completed the study could join an extension period.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Hospital Pequeno Principe
Curitiba, Brazil
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Hospital de Clinicas de Porto Alegre
Porto Alegre, Brazil
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Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira
Rio de Janeiro, Brazil
Conditions
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