News

A Republic of Congo delegation of health and higher education representatives recently met with Howard University officials, aiming to ultimately advance research into sickle cell disease (SCD) — a particularly pervasive disorder in Africa. Led by Congo Minister of Health and Population Jacqueline Lydia Mikolo and Minister of Higher…

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

Bariatric surgery  may help reduce the rates of vaso-occlusive crises (VOCs) in morbidly obese patients with sickle cell disease (SCD) without increasing the risk of mortality or other adverse events, a study says. The study, “Impact of Bariatric Surgery on Outcomes of Patients with Sickle Cell Disease: a…

Reproductive capacity, specifically the number of eggs left in the ovaries, may decline earlier in women with sickle cell disease (SCD), a study reports.  Although sickle cell disease patients in this study had no reported fertility problems, they produced less…

Brain oxygen supply is different in different regions of the brain of sickle cell disease (SCD) patients who are affected by anemia, which may explain the incidence of silent strokes, a recent study says. The findings of the study, “White matter has impaired resting oxygen delivery in sickle…

A Phase 1/2 clinical trial evaluating investigational therapy CTX001 for severe sickle cell disease (SCD) has enrolled its first patient in the U.S., and treatment is expected to begin by mid-2019. The trial (NCT03745287), still recruiting participants in Nashville, Tennessee, is looking to enroll 45 adults, 18 to 35 years old,…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

Global Blood Therapeutics (GBT) intends to improve access to high-quality healthcare for people with sickle cell disease (SCD) in the U.S. through the recently launched Access to Excellent Care for Sickle Cell Patients Pilot Program (ACCEL). The company will donate up to $150,000 to speed the development of…

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…