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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Study: ANAVEX 3-71 as a Treatment for Neurodegenerative Diseases
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Study: ANAVEX 3-71 as a Treatment for Neurodegenerative Diseases

Anavex Life Sciences Corporation has recently announced that they will conduct a clinical trial for their small molecule, ANAVEX®3-71. This therapy is intended to treat neurodegenerative and neurodevelopmental conditions, such…

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After Civil War Drove Her From Her Homeland, She Went on to Discover the Genes Causing Spinocerebellar Ataxia and Rett Syndrome
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After Civil War Drove Her From Her Homeland, She Went on to Discover the Genes Causing Spinocerebellar Ataxia and Rett Syndrome

As originally reported in Discover Magazine, Huda Zoghbi took an unexpected route to winning the Breakthrough Prize in Life Sciences in 2017 for her discoveries of the genes responsible for…

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Investigational Treatment for Rett Syndrome gets Rare Pediatric Disease Designation
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Investigational Treatment for Rett Syndrome gets Rare Pediatric Disease Designation

According to a story from Wapakoneta Daily News, the drug companies ACADIA Pharmaceuticals Inc. and Neuren Pharmaceuticals Limited have recently announced that its investigational product candidate trofinetide has earned Rare…

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These Two Companies Just Announced a Partnership to Develop Multiple Gene Therapies
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These Two Companies Just Announced a Partnership to Develop Multiple Gene Therapies

According to a story from BioSpace, the gene therapy companies Sarepta Therapeutics, Inc. and StrideBio, Inc. have recently announced the completion of a licensing and collaboration agreement that could lead…

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An Experimental Treatment for Rett Syndrome Earns Rare Pediatric Disease Designation
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An Experimental Treatment for Rett Syndrome Earns Rare Pediatric Disease Designation

According to a story from marketscreener.com, the biopharmaceutical company Anavex Life Sciences Corp. has announced that its experimental product candidate ANAVEX2-73 has earned Rare Pediatric Disease designation from the US…

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