News

Global Blood Therapeutics (GBT) is taking proactive measures to support public health efforts and sickle cell disease (SCD) patients during the global COVID-19 pandemic. To help stem the spread of the coronavirus, the company is backing public health strategies, including social distancing. Specifically, GBT is temporarily prohibiting all field…

The Institute for Clinical and Economic Review (ICER) says three recently approved therapies for managing sickle cell disease (SCD) are too costly based on early assessments of their effectiveness, and is recommending their prices be lowered. In a new evidence report, the independent watchdog agency on drug…

People with sickle cell disease (SCD) have a higher risk of bleeding from the upper portion of the gastrointestinal (GI) tract, possibly due to the use of certain anti-inflammatory medications and stress brought on by frequent hospitalizations, a study has found. The study, “…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

The American Society of Hematology released new clinical guidelines on the use of blood transfusions in people with sickle cell disease (SCD). The transfusion guidelines are the second in a series of five therapy guidelines ASH is developing for SCD. Guidelines for managing heart and kidney disease…

In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…

Record numbers of patients and advocates from across the state of Georgia — which has the nation’s fourth-highest number of people with sickle cell disease (SCD) — turned out recently for the annual Sickle Cell Day at the State Capitol. The day’s annual meeting with legislators and healthcare providers…

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…