Rare diseases are challenging. Not just for those affected, but for researchers, physicians, and drug developers. Rare diseases may affect small populations. Many are poorly understood and lack adequate awareness.…
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…
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…
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…
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…
When gene therapy company Asklepios BioPharmaceutical, Inc. ("AskBio") and biotechnology company Selecta Biosciences ("Selecta") joined forces to combat genetic disorders, each company brought its best technology and insights. AskBio…
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…
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…
Methylmalonic Acidemia Methylmalonic acidemia (MMA) is a rare disease affecting the metabolic system. 60% of all cases are due to a deficiency in MUT, a mitochondrial enzyme, which causes acids to…
According to a story from Acrofan, the biotechnology company Moderna, Inc. recently released the results of a preclinical study which described the use of mRNA which encodes a monoclonal antibody…
The National Institutes of Health (NIH) is the primary medical research institution in the United States. The NIH is one of several public health institutions, like the Food and Drug…
Methylmalonic Acidemia (MMA) is a liver condition which affects the body's ability to break down food and fatty acids. A dietary imbalance or minor virus for these patients may spark a metabolic…
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