FDA Lifts Clinical Hold on LB-001 IND for Methylmalonic Acidemia
Source: pixabay.com

FDA Lifts Clinical Hold on LB-001 IND for Methylmalonic Acidemia

During the Phase 1/2 SUNRISE clinical trial, which evaluated LB-001 for pediatric patients with methylmalonic acidemia, at least two participants experienced serious and severe adverse reactions: thrombotic microangiopathy. While investigating…

Continue Reading FDA Lifts Clinical Hold on LB-001 IND for Methylmalonic Acidemia

Don’t Miss National Eosinophil Awareness Week!

It's time to raise awareness for and celebrate the patient population that lives with eosinophilic disorders. From May 15th until the 21st, the American Partnership for Eosinophilic Disorders (APFED) is…

Continue Reading Don’t Miss National Eosinophil Awareness Week!
“New Era for Preventing End-Stage Kidney Disease Act” Could Improve Rare Disease Kidney Care
source: pixabay.com

“New Era for Preventing End-Stage Kidney Disease Act” Could Improve Rare Disease Kidney Care

The New Era for Preventing End-Stage Kidney Disease Act (H.R. 7506) is a new Act sponsored by Reps Gus Bilirakis and G.K. Butterfield. This Act would transform care for those…

Continue Reading “New Era for Preventing End-Stage Kidney Disease Act” Could Improve Rare Disease Kidney Care

Rare Disease Clinical Trials Are Essential to Help Uncover Potential Patient Solutions: Spotlight on Classic Congenital Adrenal Hyperplasia (CAH)

Acknowledgment: This story is sponsored by Neurocrine Biosciences and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted,…

Continue Reading Rare Disease Clinical Trials Are Essential to Help Uncover Potential Patient Solutions: Spotlight on Classic Congenital Adrenal Hyperplasia (CAH)