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
Acadia Pharmaceuticals Announces U.S. FDA Approval of DAYBUE ™ (trofinetide) for the Treatment of Rett Syndrome in Adult and Pediatric Patients Two Years of…
(Source: Reuters: Health)
Source: Reuters: Health - March 11, 2023 Category: Consumer Health News Source Type: news
Dallas-based Taysha Gene Therapies to receive $50M investment from Japanese pharmaceutical company
The investment will go towards Taysha's adeno-associated virus gene therapy development programs for the treatment of Rett syndrome and giant axonal neuropathy. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - October 25, 2022 Category: Pharmaceuticals Authors: DBJ staff Source Type: news
Acadia Pharmaceuticals Announces Trofinetide New Drug Application for the Treatment of Rett Syndrome has been Accepted for Filing and Review by U.S. FDA
NDA granted priority review
Prescription Drug User Fee Act action date set for March 12, 2023
SAN DIEGO–(BUSINESS WIRE)–Sep. 12, 2022– Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the U.S. Food and Drug... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - September 12, 2022 Category: Drugs & Pharmacology Source Type: news
What you should know about Rett syndrome, the condition that Richard Engel's son had
NBC correspondent Richard Engel announced that his son Henry, who had been diagnosed with Rett syndrome as an infant, died on August 9. Here's what to know about the disorder.(Image credit: Jemal Countess/Getty Images) (Source: NPR Health and Science)
Source: NPR Health and Science - August 23, 2022 Category: Consumer Health News Authors: Matt Adams Source Type: news
Study Funded by the Rett Syndrome Research Trust Demonstrates...
This week the Proceedings of the National Academy of Sciences published a paper online demonstrating that RNA editing can improve symptoms in a mouse model of Rett syndrome, a rare and severe...(PRWeb August 11, 2022)Read the full story at https://www.prweb.com/releases/2022/8/prweb18839552.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - August 11, 2022 Category: Pharmaceuticals Source Type: news
