Rheumatology

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FDA will not meet original PDUFA data for sebetralstat to treat HAE | Image Credit: Contemporary Pediatrics
FDA will not meet original PDUFA date for sebetralstat to treat HAE

June 16th 2025

Though the decision will not meet the June 17, 2025, target date, the federal agency anticipates a decision within approximately 4 weeks.

Positive data: Navepegritide, lonapegsomatropin combination treatment for children with achondroplasia | Image Credit: © lexiconimages - © lexiconimages - stock.adobe.com.
Positive data: Navepegritide, lonapegsomatropin combination treatment for children with achondroplasia

June 9th 2025

FDA accepts navepegritide application to treat children with achondroplasia | Image credit: Contemporary Pediatrics
FDA accepts navepegritide application to treat children with achondroplasia

June 2nd 2025

FDA approves Jivi for patients aged 7 years and older with hemophilia A | Image credit: Contemporary Pediatrics
FDA approves Jivi for patients aged 7 years and older with hemophilia A

May 21st 2025

Data highlights motor function improvements with ELEVIDYS treatment | Image Credit: © Colored Lights - © Colored Lights - stock.adobe.com.
Duchenne muscular dystrophy gene therapy demonstrates improved motor functions

May 16th 2025

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