This Nonprofit’s Clinical Trial Committee Aims to Help Rett Syndrome Drug Development
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This Nonprofit’s Clinical Trial Committee Aims to Help Rett Syndrome Drug Development

According to a story from news-medical.net, the International Rett Syndrome Foundation (IRSF) has announced the launch of its Clinical Trial Committee. The committee was implemented with the goal of helping…

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Stoke Therapeutics and Acadia Pharmaceuticals Collaborate to Develop Medicines for Rare Neurodevelopmental Disorders

Stoke Therapeutics and Acadia Pharmaceuticals made a joint announcement this week through Business Wire of their collaboration in the development of RNA-based medicines to potentially treat rare neurodevelopmental disorders of…

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New Genomic DNA Modification in Zebrafish Could Help Us Study Human Disease
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New Genomic DNA Modification in Zebrafish Could Help Us Study Human Disease

Did you know that zebrafish, a tropical freshwater fish, are being more commonly used to model and study human diseases? Now, says Phys.org, researchers from the Garvan Institute of Medical…

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Rett Syndrome Treatment TSHA-102 Received Rare Pediatric Disease and Orphan Drug Designations

Recently, gene therapy company Taysha Gene Therapies ("Taysha") announced that its gene therapy candidate, TSHA-102, received both Orphan Drug and Rare Pediatric Disease designations from the FDA. TSHA-102, delivered via…

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Scientists Find an Array of Genes That May Lead to Precision Therapies for Various Types of Autism

According to an article in the publication Labroots, autism spectrum disorder (ASD) may be the result of interaction between environmental and genetic factors. For instance, researchers point out that ASD…

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She Survived the Lebanese Civil War and 30 Years of Challenges to Help Rett Syndrome Patients

Huda Zoghni was born in Lebanon. Huda attended medical school at the American University located in Beirut in 1975. According to an article in Discover, Huda was forced to leave…

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Molecular Discovery Will Lead to Greater Understanding of X-Linked Genetic Diseases Like Rett Syndrome
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Molecular Discovery Will Lead to Greater Understanding of X-Linked Genetic Diseases Like Rett Syndrome

Female mammals have XX chromosomes and males have XY. But scientists have never understood how females decide which of their two X's should be active in each of its cells.…

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