Tag Archives: Rare

Chiesi Global Rare Diseases Announces FDA Approval of Ferriprox (deferiprone) for Treatment of Transfusional Iron Overload due to Sickle Cell Disease

May 1, 2021 Approval is based on demonstrated reduction in liver iron concentration Expanded indications for patients with sickle cell disease or other anemias, as well as thalassemia – BOSTON, May 1, 2021 /PRNewswire/ — Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), announced today… Read More »

Boston Red Sox chairman hopes Covid game cancellations will be a ‘rare event’ this MLB season

Boston Red Sox Chairman Tom Werner told CNBC on Thursday he does not expect coronavirus outbreaks to significantly alter the course of the Major League Baseball season. Werner’s comments on “Squawk Box” came on Opening Day, with a full slate of games on the schedule featuring all 30 teams. It marks the start of the… Read More »

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 »