News

April 1, 2020April 1, 2020

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

News

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. ... Read more

March 31, 2020March 31, 2020

Inflammation Markers Identify Sickle Cell Patients at Risk for Worse Outcomes, Study Suggests

News

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 ... Read more

March 26, 2020March 26, 2020

Availability of Endari Not Affected by COVID-19 Pandemic, Emmaus Says

News

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 ... Read more

March 25, 2020March 25, 2020

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

News

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 ... Read more

March 24, 2020March 24, 2020

GBT Supports Sickle Cell Patients During COVID-19 Pandemic

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 ... Read more

March 19, 2020March 19, 2020

Approved SCD Therapies Too Costly, ICER Watchdog Group Says

News

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, ... Read more

March 17, 2020March 17, 2020

Acupuncture May Alleviate Pain in Children and Adolescents with SCD, Study Suggests

News

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 in ... Read more

March 12, 2020March 12, 2020

Higher Risk of Upper GI Bleeds in SCD, Study Finds

News

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 ... Read more

March 10, 2020March 10, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

News

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 ... Read more

March 10, 2020March 10, 2020

Lack of Vitamin D in SCD Children Linked to Worse Hospital Outcomes in Study

News

Children and adolescents with sickle cell disease (SCD) who lack vitamin D are hospitalized more often due to pain crises, have longer hospital stays, and make more frequent visits ... Read more