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…
About BridgeBio Pharma BridgeBio Pharmaceuticals was created in 2015 by veterans from the fields of academia and biotechnology. The company focuses on the development of new therapeutic treatments for genetic…
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 People, parents Lester and Noreen Jessop from Utah recently discovered that all three of their children have a rare, life-threatening disease called pantothenate kinase-associated neurodegeneration. Their firstborn…
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)…
The Neurodegeneration with Brain Iron Accumulation (NBIA) Disorders Association was established in 1996 to serve and support all those affected by PKAN, PLAN, MPAN, BPAN, and CoPAN. Of these, PKAN or…
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