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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Trofinetide Granted Rare Pediatric Disease Designation for the Treatment of Rett Syndrome

ACADIA Pharmaceuticals, working with Neuren Pharmaceuticals, has recently been granted the Rare Pediatric Disease designation from the FDA for their therapy Trofinetide. This drug is intended for the treatment of…

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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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First Ever International Burden of Illness Survey for Rett Syndrome to be Conducted
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First Ever International Burden of Illness Survey for Rett Syndrome to be Conducted

According to a story from BioSpace, the biopharmaceutical company Newron Pharmaceuticals S.p.A. has announced that is partnering with the Rett syndrome disease community in order to sponsor an all new…

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Chief Foreign War Correspondent at NBC News Gives Account of His 4-Year-Old Son With Rett Syndrome

  Richard Engel’s son Henry, age 4, has been diagnosed with Rett syndrome, a thus far incurable genetic neurological disease. Engel is chief foreign war correspondent at NBC and has…

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Independent Data Review Gives Go-Ahead for Rett Syndrome Clinical Trial
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Independent Data Review Gives Go-Ahead for Rett Syndrome Clinical Trial

According to a story from Financial Buzz, the biopharmaceutical company Anavex Life Sciences Corp. has recently announced that the Independent Data Safety Monitoring Board (DSMB) has completed its review of…

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First Patient Dosed in Phase 2 Trial of Experimental Rett Syndrome Treatment
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First Patient Dosed in Phase 2 Trial of Experimental Rett Syndrome Treatment

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Anavex Life Sciences Corp. has just announced that the first patient has been dosed in its phase 2 clinical trial, which…

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