Children born with propionic acidemia require life-long treatment and care; without it, this disorder can lead to coma or even death. While there are supportive treatment options, no current…
According to a news release from biopharmaceutical company BridgeBio Pharma, Inc., the first patient was dosed in a Phase 1 study. Researchers are evaluating the safety, tolerability, pharmacodynamics, and pharmacokinetics…
The HealthWell Foundation is a nonprofit organization with a mission to reduce financial barriers to care for underinsured patients with chronic or life-altering diseases. The foundation assists with copays,…
HCU Network America, Organic Acidemia Association, and Propionic Acidemia Foundation's "LAND OF THE FREE, HOME OF THE BRAVE" 2021 Conference June 2022 The Land of the Free, Home of the…
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…
The National Institute of Health (NIH) has created a new approach to diagnosing and treating methylmalonic acidemia (MMA), a rare genetic disorder that stops the body from metabolizing fats and…
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