Tag Archives: Rare

Rare Pharmacy Emerges From Specialty, Part 2 – Pharmacy Times

Gordon J. Vanscoy, PharmD, MBA; Pamela Koener, PharmD; and Richard Faris, PhD, RPh Part 1 of this 3-part series, published in last month’s Directions in Oncology Pharmacy™, explored how changes in US federal policy and advances in scientific research have led to the dawn of medical breakthroughs for rare disorders. Part 2 describes how rare… Read More »

Value-based care payments increasing but risk still rare

Dive Brief: More than half of all commercial plan payments to hospitals in 2017 were earmarked toward some form of value-oriented care or alternative payments intended to reduce waste, according to the latest scorecard from Catalyst for Payment Reform (CPR) — up from just 10.2% in 2012. However, a large chunk of the value-oriented payment (90%) was… Read More »

‘I was devastated beyond belief, our whole family was’ – Irish mum opens up about her son’s rare disorder

So uncommon is his condition, that it took four years for him to be diagnosed, as many of the signs are similar to autism. But his mum says that despite the many issues he deals with, he is still a very happy little boy. “Harry has a mutation of the SOX 5 gene and when… Read More »

FDA approves Clinuvel’s rare genetic disorder treatment, shares at record high

(Reuters) – The U.S. Food and Drug Administration approved Australian drugmaker Clinuvel Pharmaceuticals Ltd’s treatment for a rare inherited disorder that causes skin damage from exposure to light, sending its shares to a record high on Wednesday. The treatment, Scenesse, is an under-the-skin implant that treats erythropoietic protoporphyria (EPP), a painful disorder that causes the… Read More »

Mother was accused of BURNING her sons after a rare condition left them with ‘butterfly skin’ 

Mother, 38, reveals police accused her of BURNING her sons after a rare condition left them with ‘butterfly skin’ Melissa Jaquez has two sons with recessive dystrophic epidermolysis bullosa Left the boys with excruciating, blistering skin that meant they could not walk People became suspicious and reported her when they were too ill for school  By Alexandra… Read More »