Rett Syndrome Research Trust Launches the MECP2 Editing Consortium With Goal to Advance Programs to Clinical Trials
The Rett Syndrome Research Trust (RSRT) has taken a significant stride in the pursuit of innovative genetic medicines for Rett syndrome by establishing the MECP2 Editing Consortium. Mutations in MECP2 cause Rett syndrome, a debilitating neurological disorder that causes a lifetime of... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - April 10, 2024 Category: Pharmaceuticals Source Type: news
PMD Solutions Supports RSRT's Biosensor VIBRANT Study for Rett Syndrome with Donation of FDA-cleared RespiraSense Devices and Analytics
The Rett Syndrome Research Trust (RSRT) is proud to announce its partnership with PMD Solutions, a leading provider of innovative healthcare technologies, in their upcoming biosensor validation study called VIBRANT. PMD Solutions will generously donate their state-of-the-art RespiraSense... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - March 28, 2024 Category: Pharmaceuticals Source Type: news
Rett Syndrome Research Trust Awards a Record $10.3 Million in 2023 to Advance a Cure for Rett Syndrome
The Rett Syndrome Research Trust (RSRT) continues to spearhead groundbreaking research in its pursuit of a cure for Rett syndrome. RSRT has announced an investment of $10.3 million in 2023 to highly innovative research projects, bringing together leading scientists, institutions, and... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - February 1, 2024 Category: Pharmaceuticals Tags: VEN Source Type: news
The Rett Syndrome Research Trust Strengthens Research Team with Addition of Robert Deans as Chief Technology Officer
The Rett Syndrome Research Trust (RSRT) is excited to announce the addition of Robert Deans, PhD, as Chief Technology Officer, Head of Research. Deans brings a wealth of genetic medicine knowledge and hands-on industry experience to RSRT. Most recently, he served as Chief Technology... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - January 16, 2024 Category: Pharmaceuticals Tags: PER Source Type: news
Why Clinical Stage Biopharmaceutical Company Anavex Shares Are Tanking Today
Anavex Life Sciences Corp. AVXL shares are trading lower in the premarket session on Tuesday after the company provided an update on Rett Syndrome program. The company reported topline results from the randomized, double-blind, placebo-controlled, Phase 2/3 EXCELLENCE clinical trial, which…#avxl #rettsyndrome #rsbq #excellence #fragilex (Source: Reuters: Health)
Source: Reuters: Health - January 2, 2024 Category: Consumer Health News Source Type: news
Rett Syndrome Research Trust Launches the Rett Syndrome Global Registry: A Pioneering Parent-Reported Platform Designed to Expedite the Development of Genetic Medicines
The Rett Syndrome Research Trust (RSRT) is excited to announce the launch of the Rett Syndrome Global Registry, a fully-remote platform for parents to share their knowledge and experiences caring for loved ones with Rett syndrome. Inclusivity is a vital component of the Rett Global... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - November 10, 2023 Category: Pharmaceuticals Tags: PDT Source Type: news
Rett Syndrome Research Trust's Genetic Medicines Summit Sets the Stage for Accelerated Therapeutic Development
The Rett Syndrome Research Trust (RSRT) is delighted to announce the successful conclusion of the inaugural Rett Syndrome Genetic Medicines Summit, held September 13 – 15 in Boston, Massachusetts. This landmark event gathered distinguished scientists, clinicians, regulatory experts, and... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - October 11, 2023 Category: Pharmaceuticals Source Type: news
What is the Diagnostic Yield for Genetic Testing of Children with Neurodevelopmental Delays?
Discussion
Children having significant neurodevelopmental disabilities are common with 17% of 3-17 year olds children having some type of such disability. Many are recommended to have genetic testing. Three main reasons for testing are determining the etiology, potential availability of targeted treatment or intervention, and determination of recurrence risk. But there are other pros and some cons to genetic testing which include:
Pros
Diagnosis is determined
Improved understanding of diagnosis, natural history and prognosis for patient and family including decreased anxiety/guilt
“Can end the diagnostic odyssey...
Source: PediatricEducation.org - July 10, 2023 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news
Why Does He Rock His Body?
Discussion
Stereotypies are “…patterned, repetitive, purposeless, involuntary movements that are also rhythmic and continual and tend to change little over time.” They occur at all times of the day, and can get worse with stress or heightened emotions including happiness. Stereotypies more often occur in children with developmental disabilities (up to 61%), sensory impairments (e.g. blindness) or social deprivation. They can be present more often in patients with autism (up to 88%). They can be seen in ~7% of normally developing children too. Their onset is before age 3 years and tend to improve over year...
Source: PediatricEducation.org - May 22, 2023 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news
Drug for rare condition tested at UAB, gets FDA approval
The drug is marketed as the first treatment for Rett syndrome. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - April 10, 2023 Category: Pharmaceuticals Authors: Laurel Thrailkill Source Type: news
FDA OKs First Drug for Rett Syndrome FDA OKs First Drug for Rett Syndrome
Trofinetide (Daybue) is for adults and children aged 2 years and older with Rett syndrome, a rare, genetic neurodevelopmental disorder affecting mostly females.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - March 14, 2023 Category: Neurology Tags: Neurology & Neurosurgery News Alert Source Type: news
FDA Approves Daybue, the First Treatment for Rett Syndrome
TUESDAY, March 14, 2023– The U.S. Food and Drug Administration has approved Daybue (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients aged 2 years and older.
Approval was based on results from the phase 3... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - March 14, 2023 Category: General Medicine Source Type: news
FDA Approves Daybue (trofinetide) for the Treatment of Rett Syndrome
SAN DIEGO–(BUSINESS WIRE)–Mar. 10, 2023– Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the U.S. Food and Drug Administration (FDA) has approved Daybue (trofinetide) for the treatment of Rett syndrome in adult and... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - March 13, 2023 Category: Drugs & Pharmacology Source Type: news
FDA approves first treatment for Rett Syndrome
FDA has approved Daybue (trofinetide) oral solution as the first treatment for Rett syndrome, a rare, genetic neurological disorder. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - March 13, 2023 Category: Drugs & Pharmacology Authors: FDA Source Type: news
First Treatment Approved for Rare Genetic Neurologic Disorder
(MedPage Today) -- The FDA approved trofinetide (Daybue), the first drug to treat adult and pediatric patients age 2 and older with Rett syndrome, drugmaker Acadia Pharmaceuticals announced Friday.
"Now, for the first time after decades of clinical... (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - March 13, 2023 Category: Neurology Source Type: news
