The Medical College of Georgia (MCG) and Augusta University Health (AU Health) have joined the first large, national clinical trial investigating bone marrow transplantation as part of the standard of care (SoC) for patients with sickle cell disease (SCD). The National Institutes of Health (NIH)-funded study will…
News
Hydroxyurea, a treatment recommended for children with sickle cell anemia (SCA), doesn’t increase the risk of malaria infection in sub-Saharan African countries where malaria is endemic, clearing up previous suspicions, a new study finds. Sold under the brand names Hydrea (Bristol-Myers Squibb) or Droxia (Bristol-Myers Squibb),…
Cydan, the company that founded Imara — which developed a sickle cell disease (SCD) treatment candidate, IMR-687 — has raised $34 million in new financing to advance innovative therapies for patients living with SCD and other rare diseases. Cydan, based in Cambridge, Massachusetts, identifies promising scientific findings and drug…
More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…
The California Institute for Regenerative Medicine (CIRM) awarded Matthew Porteus, a Stanford University associate professor of pediatrics, a $5.2 million grant to conduct the groundwork leading to a clinical trial of a possible treatment for sickle cell disease (SCD). SCD develops when a gene is mutated that codes for a…
News
#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives it offers pharmaceutical companies to develop therapies for rare diseases — off the table. That’s the message being pushed by the National Organization for…
The Medical University of South Carolina (MUSC) was recently awarded a Doris Duke Charitable Foundation grant to develop a new gene-modifying therapy for the treatment of sickle cell disease. The project, “Epigenetic Modulators for the Treatment of Sickle Cell Disease,” was awarded more than $750,000 as part of the…
The U.S. Food and Drug Administration (FDA) has awarded Dr. Kenneth Ataga, a $2 million grant over five years to support a prospective study in sickle cell anemia to determine biomarkers of specific cellular function changes in chronic kidney disease (CKD). Ataga, a researcher with the University of…
Researchers have found that patients with sickle cell disease (SCD) have increased risk of developing leukemia, but lower risk for breast cancer or male genital cancers. These findings resulted from a population-based study titled “Increased risk of leukemia among sickle cell disease patients in California” that was published in…
Sancilio Pharmaceuticals has completed a Phase 2 clinical trial evaluating Altemia (docosahexaenoic acid) as a treatment for children aged 5 to 17 with sickle cell disease (SCD). The company expects to release the main results of the SCOT trial (NCT02973360) by the end of 2017. The study assessed…
Recent Posts
- Adults with SCD face high risk of stroke in their 30s and 40s, study finds
- Sickle cell doctors face higher burnout than other specialists: Study
- Disease burden heavy for SCD patients in US, despite promise of gene therapy
- The conversation we avoid: Telling children they have sickle cell disease
- New study finds distinct molecular signatures in newborns with SCT
- Blood test may improve kidney damage detection in children with SCD
- Understanding the impact of leg ulcers in sickle cell disease
- Asthma seven times more likely in SCD children than in unaffected siblings
- Sickle cell drug at high dose eases anemia symptoms in severe SCD
- Researchers urge more talk on menstrual pain in sickle cell clinics