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Rett Syndrome

All articles tagged with #rett syndrome

health4 hours ago•9 min saved

EMA CHMP June 2026: six medicines advance, Tavneos revoked, and broad indication extensions

At its June 2026 meeting, the CHMP recommended six medicines for approval (Aujemflu influenza vaccine for adults 50+, Hopledo for Parkinson’s, Onswik for type 2 diabetes) and two biosimilars (Denosumab Ascend, Nylaspeg); Daybu passed re-examination with a restricted Rett syndrome indication. It issued negative opinions for Tacquell, Yartemlea and Xervyteg. The CHMP also endorsed extensions of indication for 12 medicines (including Imvanex to ages 2+, and Datroway, Jaypirca, Leqvio, MenQuadfi, Opzelura, Orladeyo, Rezolsta, Rinvoq, Stelara, Symtuza, Tecvayli). Withdrawals were made for DuoResp Spiromax and BiResp Spiromax extensions. Start of referral on Rifadin due to excipient concerns; Tavneos’ marketing authorisation was revoked after review. Other updates include Fluenz can be administered by non-healthcare professionals under supervision, Ixchiq extension to high-risk 12+, and including Mounjaro data in product information without a new indication. CHMP statistics noted 6 positive opinions on new medicines, 3 negative opinions, 13 positive opinions on extensions, and 0 withdrawals for new medicines this year.

via European Medicines Agency (EMA)|

#chmp #drug-approval #ema

health4 months ago•34 min saved

Normal for 18 months, then Rett syndrome: a family's long road to a diagnosis

A father recounts how his daughter appeared perfectly normal for the first 18 months, then rapidly regressed in speech and motor skills, leading to a long diagnostic journey that culminated in Rett syndrome in October 2022; after initial ER visits and a misdiagnosis of autism, genetic testing confirmed Rett, and now five-year-old Talia receives multiple therapies with the family hopeful for FDA-approved treatment and continued support.

via Daily Mail|

#developmental-regression #diagnosis-journey #genetic-disorder

health3 years ago•3 min saved

FDA Releases Latest Updates on March 14, 2023.

The FDA has revised the Janssen COVID-19 Vaccine Fact Sheet to include warnings about increased risks of myocarditis, pericarditis, and facial paralysis following vaccination. The scope of authorization for a booster dose has also been revised. The FDA has approved Daybue as the first treatment for Rett syndrome in adults and children. There are now 444 COVID-19 tests and sample collection devices authorized by the FDA under emergency use authorizations.

via FDA.gov|

#covid-19-testing #covid-19-vaccine #fda