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Emerging Therapies for OTC Deficiency Show Continued Progress
Summary: This article is presented in English. The initial articles come from BioSpace and Fierce Biotech. Cet article est presente en anglais. Les articles initiaux proviennent de BioSpace et Fierce Biotech.

Satellite Bio Announces FDA Rare Pediatric Disease Designation for SB-101 for the Treatment of Urea Cycle Disorders (UCDs)
Summary: This article is presented in English. The article comes from Satellite Bio. Cet article est presente en anglais. L'article provient de Satellite Bio.

CDA-AMC Reimbursement Review: Patient and Clinician Group Input for pegzilarginase (ARG1-D)
Summary: This article is presented in English. The article comes from Canada's Drug Agency (CDA-AMC). Cet article est presente en anglais. L'article provient de l'Agence des medicaments du Canada (ACMTS / CDA-AMC).

QU’s genetic discovery offers new hope for treating Homocystinuria
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.

Aurora Therapeutics Launches to Realize Potential of Personalized Gene Editing for Millions of Patients with Rare Diseases
Summary: This article is presented in English. The article comes from Aurora Therapeutics. Cet article est presente en anglais. L'article provient d'Aurora Therapeutics.

Une histoire de PCU | Nathan et la phénylcétonurie: vivre avec une maladie invisible au quotidien
Summary: Note en francais : Cet article est presente en francais. L'article provient de phenylcetonurie.ca. Auteur : Tristan Audet. English note: This article is presented in French. The article comes from phenylcetonurie.ca. Author: Tristan Audet.

A PKU story | Nathan and phenylketonuria: living with an invisible disease on a daily basis
Summary: This article is presented in English. The article comes from phenylcetonurie.ca. Author: Tristan Audet. Cet article est presente en anglais. L'article provient de www.phenylcetonurie.ca.

Sephience, nouveau traitement pour la phénylcétonurie, est approuvé au Canada :
Summary: Cet article est presente en francais. L'article provient de Carnet Phénylcétonurie par Tristan. This article is presented in French. The article comes from Carnet Phénylcétonurie by Tristan.

Genome sequencing pilot tests for UCDs
Summary: This article is presented in English. The article comes from News-Medical.net. Cet article est présenté en anglais. L'article provient de News-Medical.net.

Application of high-resolution mass spectrometry profiling towards the diagnosis and acute management of maple syrup urine disease
Summary: This article is presented in English. The article comes from PubMed. Authors/Auteur: Rafael Garrett, Sara Pickett, Melinda J Peters, Khadija Belhassan, Adam S Ptolemy, Roy W A Peake Cet article est presente en anglais. L'article provient de PubMed.