A gene editing technology called CRISPR/Cas9 repaired 20 to 40 percent of stem and progenitor cells obtained from sickle cell disease patients, scientists report, suggesting such an approach might offer a way of treating this disease. Sickle cell anemia is the most severe type of sickle cell disease, which is…
At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated 30 million Americans with rare diseases. Rep. Leonard Lance (R-New Jersey) and Sen. Amy Klobuchar (D-Minnesota) spoke to more…
An upcoming Phase 1 clinical trial was recently awarded $5.74 million to evaluate an innovative blood stem cell transplant procedure for adults with severe sickle cell disease (SCD). The California Institute for Regenerative Medicine (CIRM) granted the funding to City of Hope, where the research will be conducted, and…
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…
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…
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