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About 2,110 results

ALLMedicine™ Rett Syndrome Center

Research & Reviews  801 results

Contribution of Mendelian disorders in a population-based pediatric neurodegeneration c...
https://doi.org/10.1016/j.jpeds.2022.05.017
The Journal of Pediatrics; Guo R, Mentch FD et. al.

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...

Anxiety-like behavior and anxiolytic treatment in the Rett syndrome natural history study.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9107202
Journal of Neurodevelopmental Disorders; Buchanan CB, Stallworth JL et. al.

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...

ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome
https://clinicaltrials.gov/ct2/show/NCT04304482

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 ...

Bringing machine learning to research on intellectual and developmental disabilities: t...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9059371
Journal of Neurodevelopmental Disorders; Gupta C, Chandrashekar P et. al.

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...

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Guidelines  1 results

Guidelines for management of scoliosis in Rett syndrome patients based on expert consen...
https://doi.org/10.1097/BRS.0b013e3181a95ca4
Spine Downs J, Bergman A et. al.

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 ...

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Clinicaltrials.gov  19 results

ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome
https://clinicaltrials.gov/ct2/show/NCT04304482

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 ...

The Rett Syndrome Global Registry
https://clinicaltrials.gov/ct2/show/NCT04900493

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...

Open-Label Extension Study of Trofinetide for Rett Syndrome
https://clinicaltrials.gov/ct2/show/NCT04776746

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

Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
https://clinicaltrials.gov/ct2/show/NCT04181723

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

Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
https://clinicaltrials.gov/ct2/show/NCT03848832

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.

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News  20 results

Novel Drug Offers New Hope for Rett Syndrome
https://www.medscape.com/viewarticle/971720

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...

Reducing Pain Medication Not a Factor in Rett Syndrome Girl's Death: Coroner
https://www.medscape.com/viewarticle/948519

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 ...

BRIEF-Anavex Life Sciences Announces Exceeding Of Enrollment Target For Anavex2-73 U.S. Phase 2 Rett Syndrome Clinical Trial
https://www.reuters.com/article/brief-anavex-life-sciences-announces-exc/brief-anavex-life-sciences-announces-exceeding-of-enrollment-target-for-anavex2-73-u-s-phase-2-rett-syndrome-clinical-trial-idUSASA00TW6

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...

BRIEF-Acadia Pharmaceuticals And Neuren Pharmaceuticals Announce Rare Pediatric Disease Designation For Trofinetide
https://www.reuters.com/article/brief-acadia-pharmaceuticals-and-neuren/brief-acadia-pharmaceuticals-and-neuren-pharmaceuticals-announce-rare-pediatric-disease-designation-for-trofinetide-idUSFWN2AW0VB

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...

Novel Drug Promising for Life-Threatening Rett Syndrome in Kids
https://www.medscape.com/viewarticle/911519

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...

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Patient Education  3 results see all →