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.…
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…
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…
According to a story from the Bensalem Times, David and Catherine Woodley's adopted daughter Abby recently underwent a transplant operation that saved her life. David and Catherine adopted Abby three…
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