ALLMedicine™ Rett Syndrome Center

May 17th, 2022 - To evaluate Mendelian causes of neurodegenerative disorders in a cohort of pediatric patients. Patients enrolled in the Center for Applied Genomics (CAG) Biobank at the Children's Hospital of Philadelphia with neurodegenerative symptoms were ident...

May 16th, 2022 - Rett syndrome (RTT) is a neurodevelopmental disorder most often related to a pathogenic variant in the X-linked MECP2 gene. Internalizing behaviors appear to be common, but standard methods of diagnosing anxiety are not readily applied in this pop...

May 9th, 2022 - This Phase 2/3 efficacy study is designed as a double-blind, randomized, placebo-controlled study. This is a 12-week placebo-controlled study of ANAVEX2-73 oral solution for the treatment of patients with RTT 5-17 years of age. A voluntary option ...

May 4th, 2022 - Intellectual and Developmental Disabilities (IDDs), such as Down syndrome, Fragile X syndrome, Rett syndrome, and autism spectrum disorder, usually manifest at birth or early childhood. IDDs are characterized by significant impairment in intellect...

Apr 29th, 2022 - To identify the clinical features correlating with the presence and severity of scoliosis in girls with Rett syndrome (RTT). Seventy-five girls with a clinical and genetically determined diagnosis of RTT participated in this cross-sectional study....

Aug 1st, 2009 - Modified Delphi technique. To develop guidelines for the clinical management of scoliosis in Rett syndrome through evidence review and consensus expert panel opinion. Rett syndrome is a rare disorder and clinical expertise is thus with small case ...

May 9th, 2022 - This Phase 2/3 efficacy study is designed as a double-blind, randomized, placebo-controlled study. This is a 12-week placebo-controlled study of ANAVEX2-73 oral solution for the treatment of patients with RTT 5-17 years of age. A voluntary option ...

Apr 26th, 2022 - The Rett Syndrome Global Registry is a fully remote, global, caregiver-reported registry intended to meet the needs of caregivers, clinicians and researchers, and therapeutic developers with the goal to increase our understanding of this rare diso...

Apr 20th, 2022 - To investigate the safety and tolerability of continued long-term treatment with oral trofinetide in girls and women with Rett syndrome

Feb 17th, 2022 - To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome

Dec 29th, 2021 - To evaluate the efficacy of cannabidiol oral solution (GWP42003-P, CBD-OS) in reducing symptom severity when compared with placebo, in participants with Rett syndrome.

Apr 6th, 2022 - Trofinetide (Acadia Pharmaceuticals) is effective in improving symptoms in girls and young women with Rett syndrome (RTT), a rare and devastating neurodevelopmental disorder, results from a phase 3 study suggest. Dr Jeffrey Neul "In a severe disor...

Apr 1st, 2021 - The death of a seriously ill 11-year-old girl who had a "complicated medical history" was not contributed to by a reduction in her pain medication, a coroner has said. Melody Driscoll/PA Media She was in the headlines in 2016 when she was visited ...

Jun 16th, 2020 - June 16 (Reuters) - Anavex Life Sciences Corp: * ANAVEX LIFE SCIENCES ANNOUNCES EXCEEDING OF ENROLLMENT TARGET FOR THE ANAVEX®2-73 (BLARCAMESINE) U.S. PHASE 2 RETT SYNDROME CLINICAL TRIAL * ANAVEX LIFE SCIENCES - EXPECTS TO ANNOUNCE TOPLINE RESULT...

Mar 3rd, 2020 - March 3 (Reuters) - Neuren Pharmaceuticals: * ACADIA PHARMACEUTICALS AND NEUREN PHARMACEUTICALS ANNOUNCE RARE PEDIATRIC DISEASE DESIGNATION FOR TROFINETIDE FOR THE TREATMENT OF RETT SYNDROME * NEUREN PHARMACEUTICALS - RESULTS FROM PHASE 3 LAVENDER...

Apr 8th, 2019 - A new medication for the core signs and symptoms of Rett Syndrome (RTT), a life-threatening, neurodevelopmental condition, shows promise in children and adolescents, results of a new phase 2 study suggest. Findings from the randomized controlled t...