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
Acadia Pharmaceuticals Submits New Drug Application to the U.S. FDA for Trofinetide for the Treatment of Rett Syndrome
SAN DIEGO–(BUSINESS WIRE)–Jul. 18, 2022– Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the company has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for trofinetide for the... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - July 18, 2022 Category: Drugs & Pharmacology Source Type: news
Neurogene ’s Gene Replacement Program is the Eighth Biopharmaceutical...
The research and resource development efforts of the Rett Syndrome Research Trust (RSRT) have helped generate an eighth biopharmaceutical program that tackles the root cause of Rett syndrome....(PRWeb May 19, 2022)Read the full story at https://www.prweb.com/releases/2022/5/prweb18686581.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - May 19, 2022 Category: Pharmaceuticals Source Type: news
Novel Drug Offers New Hope for Rett Syndrome Novel Drug Offers New Hope for Rett Syndrome
New study findings offer hope for girls with this rare and devastating condition.Medscape Medical News (Source: Medscape Neurology and Neurosurgery Headlines)
Source: Medscape Neurology and Neurosurgery Headlines - April 6, 2022 Category: Neurology Tags: Neurology & Neurosurgery News Source Type: news
Trofinetide Shows Promise in Rett Syndrome
(MedPage Today) -- The investigational drug trofinetide showed modest benefit in girls and young women with Rett syndrome, the phase III LAVENDER study showed.
After 12 weeks of treatment, significant differences in two co-primary endpoints and... (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - April 6, 2022 Category: Neurology Source Type: news
When Can Menstrual Suppression Start in Patients with a Disability?
Discussion
Menstrual suppression using hormonal therapy is commonly used for a variety of reasons:
Patient preference
Contraception
Heavy or painful menses
Treatment of concomitant problems such as menstrual migraine, mood issues, nausea
Specific patient populations
Disabilities
Oncology
Transgender and gender nonbinary
Methods of suppression include:
Estrogen and progesterone
Combined oral contraceptives (COC) – very good menstrual suppression and generally used as first line treatment for many patients
Traditional dosing with active medication for 21-24 days and then 4-7 day placebo break.
Extended cycles &...
Source: PediatricEducation.org - February 14, 2022 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news
