On November 27, biotechnology company Vertex Pharmaceuticals Inc. ("Vertex") announced the approval of SYMKEVI with KALYDECO for the treatment of pediatric patients with cystic fibrosis (CF), says GuruFocus. The…
As reported in Warta Saya; for kids with cystic fibrosis (CF), a daily exercise needed to clear the lungs of the disease's characteristic build-up of mucus can feel long and…
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…
As of November 20, Zokinvy has been approved by the FDA for the treatment of Hutchinson-Gilford progeria syndrome (progeria) and a few other progeroid laminopathies in patients aged one year…
Bulgarian cystic fibrosis patient joins international campaign standing up for the rights of patients to access essential medicines Rositsa Malinova, a 22-year-old resident of Pazardzhik, Bulgaria, is taking on the…
According to a story in Charcot-Marie-Tooth News, the CMT&Me study has launched a competition in Europe with the purpose of raising awareness and money for CMT and related disorders. Any…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In many areas of society, from healthcare to big business, companies are turning to artificial intelligence (AI) to solve pressing problems. Within the medical sphere, researchers recently developed an automated,…
From December 1-4, 2020, Reuters hosted a virtual conference called Pharma & Patient USA 2020. The primary focus of this event was furthering patient centricity in disease development as well…
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