Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…
News
Tevin Coleman brought the snow inside last winter. Coleman, a running back for the New York Jets, his wife Akilah Coleman, and twins celebrated Christmas in January, to accommodate the last few weeks of the NFL regular season. His daughter, Nazaneen, 4, has sickle cell disease, and cold weather…
Two mouse models commonly used to study sickle cell disease (SCD) are not suitable for testing certain gene editing treatment approaches, including those for fetal hemoglobin production, because of their specific genetic makeup, a study reported. “This work provides a reminder for scientists to carefully consider the genetics of…
A novel strategy called Engineered Stem Cell Antibody Paired Evasion (ESCAPE) could be used to more safely prepare people with sickle cell disease (SCD) for experimental gene-editing approaches for treating the inherited blood disorder, according to researchers. ESCAPE may help patients ready for a bone marrow transplant, a key…
A public policy alliance has launched to advocate for federal policies that address the “crisis of care” facing the approximately 100,000 sickle cell disease (SCD) patients in the U.S. The ad-hoc alliance, called the Sickle Cell Disease Partnership, comprises more than a dozen groups representing patient advocates, healthcare…
Lowering the risk of long-term complications, including organ damage and hemolytic anemia — driven by the destruction of red blood cells — are common goals for sickle cell disease (SCD) patients, caregivers, and healthcare professionals, according to data from Global Blood Therapeutics’ international SHAPE survey. Survey results also…
Global Blood Therapeutics (GBT) has begun a Phase 2/3 trial to study the safety, tolerability, effectiveness, and pharmacological properties of its experimental oral therapy GBT601 in people with sickle cell disease (SCD). Initial data from the study (NCT05431088) are expected before the end of the year, according…
Precision BioSciences and Novartis are teaming up to develop and commercialize a custom gene therapy as a potential treatment approach for certain inherited blood disorders, including beta thalassemia and sickle cell disease (SCD). Under their new exclusive license and collaboration agreement, the companies will combine Precision’s proprietary ARCUS genome…
A comprehensive integrated sickle cell disease (SCD) clinic in southern California — funded by a $500,000 state grant — has been established to support the “highly affected” SCD patient population in San Bernardino County, according to a press release. The community clinic is housed at Loma Linda University…
Aflac, a U.S. supplemental health insurance company, has given $100,000 to Children’s National Hospital in Washington, D.C., to support research into sickle cell disease (SCD) and its treatments. The donation was made to honor World Sickle Cell Awareness Day, observed each June 19. “We are honored for Aflac’s partnership to…
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