New drug offers hope for families battling rare, devastating brain diseases
NCT ID NCT07399704
Summary
This study aims to gather long-term safety and effectiveness data for the drug nizubaglustat in people with rare, progressive brain disorders (GM2 gangliosidosis or Niemann-Pick type C). It includes two groups: patients continuing from a previous study and patients switching from an existing treatment. The goal is to see if the drug can help control these diseases over time.
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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Associação Hospitalar de Prot à Infância Dr. Raul Carneiro
RECRUITINGÁgua Verde, Curitiba, 80250-060, Brazil
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital de Clinicas de Porto Alegre
RECRUITINGPorto Alegre, Rio Grande do Sul, 90035-903, Brazil
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira
NOT_YET_RECRUITINGRio de Janeiro, 22250, Brazil
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Conditions
Explore the condition pages connected to this study.