Rheumatology

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Navepegritide NDA submitted to treat children with achondroplasiaLatest revision | Image Credit: © Araki Illustrations - © Araki Illustrations - stock.adobe.com.
Navepegritide NDA submitted to treat children with achondroplasia

March 31st 2025

Submission is based on 3 clinical trials, including data from the ApproaCH Trial among children with achondroplasia.

FDA approves fitusiran for hemophilia A, B patients aged 12 years and upLatest revision | Image Credit: © Calin - © Calin - stock.adobe.com.
FDA approves fitusiran for hemophilia A, B patients aged 12 years and up

March 28th 2025

FDA approves diazoxide choline extended-release tablets for Prader-Willi syndrome | Image Credit: © Calin - © Calin - stock.adobe.com.
FDA approves diazoxide choline extended-release tablets for hyperphagia in Prader-Willi syndrome

March 26th 2025

Craig McDonald, MD, breaks down positive SGT-003 data for Duchenne muscular dystrophy | Image credit: UC Davis Health
Craig McDonald, MD, breaks down positive SGT-003 data for Duchenne muscular dystrophy

March 26th 2025

Puzzler poll: Urticarial rash in a 9-hours-old male | Image credit: Provided by case authors
Puzzler poll: Urticarial rash in a 9-hours-old male

February 20th 2025

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