News

Global Blood Therapeutics (GBT) is giving $250,000 to support the U.S. sickle cell disease (SCD) community during the COVID-19 pandemic. Its newly created GBT Community Fund will award a total of $150,000 in grants to non-profit organizations supporting the needs of  SCD patients and their families during…

The U.S. Food and Drug Administration (FDA) has cleared the start of a Phase 1/2 clinical trial testing a genome editing-based therapy, known as OTQ923, in adults with severe complications of sickle cell disease (SCD). OTQ923, developed by Novartis and Intellia Therapeutics, uses the CRISPR/Cas9 genome-editing technology.

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

High blood levels of two pro-inflammatory molecules, called interleukin-6 (IL-6) and interleukin-8 (IL-8), are linked to more frequent complications in people with sickle cell anemia, a study reports. The study, “High levels of proinflammatory cytokines IL-6 and IL-8 are associated with a poor clinical outcome…

The supply of the sickle cell disease (SCD) medication Endari (L-glutamine) will not be affected by the ongoing COVID-19 outbreak, Emmaus Life Sciences said in an update to the SCD community While closely monitoring the pandemic, the company said its supply of Endari is large…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

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…

Acupuncture may be a useful add-on therapy to help manage pain in children and adolescents who have sickle cell disease, a new study suggests. The study, “Acupuncture for pain management…