Summary: Health Canada’s new Canadian Clinical Trial Search Portal makes it easier to find clinical trials authorized in Canada by condition, treatment, location, recruitment status and more. For people living with rare metabolic disorders and their families, it provides a simpler way to discover research opportunities taking place closer to home.

Summary: Pegtibatinase is an investigational enzyme replacement therapy being studied for classical homocystinuria caused by cystathionine beta synthase deficiency. The clinical development program includes the COMPOSE, HARMONY and ENSEMBLE studies, with HARMONY currently evaluating the therapy in a pivotal Phase 3 trial.

Summary: RareKids-CAN is inviting youth with rare diseases and caregivers to share how they search for and use medication-related information. The study aims to identify gaps in current resources and help shape clearer, more useful pharmacology education for rare-disease families across Canada.

Summary: A rare diagnosis is important information, but it is not the whole story. Use CanChild’s F-Words for Child Development to introduce your child’s strengths, relationships, interests and hopes to their school team.

Summary: Un diagnostic rare est une information importante, mais il ne raconte pas toute l'histoire. Utilisez les « Mes mots préférés » de CanChild pour présenter à l'équipe scolaire les forces, les relations, les intérêts et les aspirations de votre enfant.

Summary: This article is presented in English. The article comes from Tribune News Network, Doha. Cet article est presente en anglais. L'article provient de Tribune News Network, Doha. Researchers from Qatar University (QU) published a study examining a specific genetic mutation called T236N in the CBS (cystathionine beta-synthase) enzyme.