Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare…
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Gamida Cell recently presented updated data from its ongoing Phase 1/2 clinical trial on an experimental alternative to bone marrow transplants in patients with sickle cell disease (SCD). Elbit Imaging, of which Gamida is an indirect shareholder, announced the information at the 2018 BMT Tandem Meetings in…
In recognition of Rare Disease Day 2018, Bionews Services — which publishes this website — will attend and report on three relevant conferences in the U.S. dealing with policies and programs of importance to patients and their families. The three are among 50 events in 32 states…
A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and Mumbai, India. From Athens to Atlanta, from San Diego to Sydney, people across the globe will mark World Rare Disease…
Despite an increased risk of invasive pneumococcal disease (IPD) in children with sickle cell anemia (SCA), preventive treatment (prophylaxis) rates remain low among these patients, a study shows. The research, “Antibiotic Prophylaxis for Children With Sickle Cell Anemia,” was published in Pediatrics, the official journal of the…
Imara, a pharmaceutical company developing new therapies for sickle cell disease (SCD), has successfully dosed the first patient enrolled in its clinical trial evaluating escalating doses of IMR-687 in adult patients with SCD. The Phase 2 trial (NCT03401112) is currently recruiting participants and expects to enroll…
David Curtis Glebe, a retired 64-year-old public prosecutor now living in Millsboro, Delaware, knows he’s lucky to be alive. In mid-2013, while in Arizona, Glebe was diagnosed with pancreatic neuroendocrine cancer (PNET) — the same disease that killed Apple’s founder and CEO Steve Jobs. After three years of progress…
Hypersensitivity to allergens in children with sickle cell anemia may increase the risk of acute chest syndrome, a study suggests. The study “Aeroallergen sensitization predicts acute chest syndrome in children with sickle cell anaemia” was published in the British Journal of Haematology. Diagnosing asthma in children with sickle cell…
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Two Types of CRISPR Gene Editing Could Be Useful in Treating Sickle Cell Anemia, Editas Reports
Two kinds of CRISPR gene editing therapies show promise as treatments for a number of blood diseases, including sickle cell anemia, Editas Medicine reports. Gene editing is one of a wave of new approaches to treating diseases. It involves using specialized proteins to make precise cuts and pastes of DNA to correct…
Researchers found that treatment with hydroxyurea at currently approved doses for sickle cell anemia leads to a significant drop in total sperm count in men. The study, “Adverse effect of hydroxyurea on spermatogenesis in patients with sickle cell anemia after six months of treatment,” was published in the…
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