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March 19, 2019March 19, 2019

Oral Morphine is a Viable Treatment Option for SCD Patients Experiencing VOCs, Study Says

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Oral morphine treatment is a valid therapy option for patients with sickle cell disease (SCD) who are undergoing a vaso-occlusive crisis (VOC), researchers suggest. It was found to significantly reduce ... Read more

March 15, 2019March 14, 2019

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

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Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of ... Read more

March 14, 2019March 14, 2019

Congo Delegation Visits Howard University to Bolster Collaborations on Sickle Cell Disease

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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 ... Read more

March 13, 2019March 13, 2019

NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

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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 ... Read more

March 12, 2019March 12, 2019

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

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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 ... Read more

March 12, 2019March 12, 2019

Weight-loss Surgery May Help Reduce VOCs in Morbidly Obese SCD Patients

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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 ... Read more

March 7, 2019March 7, 2019

Women with Sickle Cell Disease May Have Earlier Decline of Ovarian Function, Study Suggests

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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 ... Read more

March 5, 2019March 5, 2019

Differences in Brain Oxygen Supply May Explain Silent Strokes in SCD Patients

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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, ... Read more

February 28, 2019February 28, 2019

First Patient Enrolled in Phase 1/2 Trial Testing CTX001 for Severe Sickle Cell Disease

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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 ... Read more

February 26, 2019February 26, 2019

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

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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 ... Read more

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