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

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New Technology Found To Be Better Treatment for Hereditary Tyrosinemia Type 1 and Wilson Disease

LogicBio Therapeutics has just presented preclinical data of their GeneRide platform. This data demonstrates that the GeneRide technology is valuable in methylmalonic acidemia (MMA), hereditary tyrosinemia type 1 (HT1), and…

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New Investigative Therapy for Methylmalonic Acidemia and Propionic Acidemia is Making Regulatory Progress
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New Investigative Therapy for Methylmalonic Acidemia and Propionic Acidemia is Making Regulatory Progress

Fast Track Designation and Rare Pediatric Disease Designation have just been awarded from the FDA to HemoShear Therapeutics. This designation was given to their new investigative therapy for propionic acidemia…

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Company Receives Clearance to Conduct Trial for Propionic and Methylmalonic Acidemia
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Company Receives Clearance to Conduct Trial for Propionic and Methylmalonic Acidemia

According to a press release from HemoShear Therapeutics, the company has recently received official clearance from the US Food and Drug Administration (FDA) in regards to its Investigational New Drug…

Continue Reading Company Receives Clearance to Conduct Trial for Propionic and Methylmalonic Acidemia