The Binder family never expected that their 12-year-old twins, Grady and Jace, would be diagnosed with a rare genetic disorder. In fact, this particular disorder is so rare that it…
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…
by Lauren Taylor from In The Cloud Copy Pantothenate kinase-associated neurodegeneration or PKAN is a rare, inherited condition in which the affected child experiences progressive degeneration in certain parts of…
According to a story from BioSpace, the biopharmaceutical company Retrophin, Inc., recently released the topline results from its phase 3 clinical trial. This clinical trial was testing the company's drug…
According to a story from ScienceBlog, the results of an international research study which was led by a team of scientists in Oakland, California and Munich, Germany, has led to…
Pantothenate kinase-associated neurodegeneration (PKAN) is a neurodegenerative disorder caused by a mutated PANK2 gene. This mutation disrupts a metabolic pathway, decreasing the body's levels of CoA. CoA, or coenzyme A,…
According to a story from Pet Age, a combination of business leaders, pet owners, and reptile hobbyists came together and raised $45,000 for the grieving family of Anna Hupp, who…
A biopharmaceutical company based in California called Retrophin recently announced that the first patient has been administered treatment in their phase III study in to test RE-024 (fosmetpantotenate) as a…
8-year-old Carter Anderson has PKAN. He’s only had his official diagnosis for about a year, as his parents were first told he had cerebral palsy. Since Pantothenate Kinase-Associated Neurodegeneration (PKAN)…
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