News

February 13, 2020February 13, 2020

Pediatric SCD Patients with Acute Kidney Injury Face More Hospital Care, Higher Costs, Large–scale Analysis Finds

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Pediatric patients with sickle cell disease complicated by acute kidney injury (AKI) were more likely to be admitted to the intensive care unit (ICU), had longer hospital stays, accumulated ... Read more

February 11, 2020February 11, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

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The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous ... Read more

February 11, 2020February 11, 2020

Hospital Readmissions High in Sickle Cell Patients, Study Shows

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More than a quarter of patients with sickle cell disease (SCD) are readmitted to the hospital within 30 days of being discharged, most often due to sickle cell crisis, ... Read more

February 6, 2020February 6, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

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The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over ... Read more

February 6, 2020February 6, 2020

Most U.S. Residents Support Human Genetics Research, Online Poll Says

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A recent survey found that U.S. residents are largely interested in and support continued research into the role of human genetics in treating and possibly curing illnesses such as ... Read more

February 4, 2020February 4, 2020

SCD Patients Receiving Hydroxyurea in the U.S. Still Face Many Challenges

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People with sickle cell disease (SCD) treated with hydroxyurea in the U.S. still face many challenges, including the financial burden posed by their healthcare expenses, low treatment adherence and ... Read more

January 30, 2020January 30, 2020

FDA-Approved Therapies for Sickle Cell Disease May Not be Cost-Effective, ICER Draft Report Says

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FDA-approved therapies for sickle cell disease (SCD) may not be cost-effective at their current prices, according to a draft report issued by the Institute for Clinical and Economic Review ... Read more

January 28, 2020January 28, 2020

Jasper Raises More Than $50M to Advance Its Potentially Safer Conditioning Therapy for Stem Cell Transplant

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Jasper Therapeutics has raised an additional $14.1 million to finance the development of its investigational conditioning therapy, JSP191, as a safer and more effective treatment to prepare patients for ... Read more

January 23, 2020January 23, 2020

FDA Names Potential Gene Therapy for Sickle Cell an ‘Orphan Drug,’ Aruvant Announces

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A potential gene therapy for sickle cell disease (SCD) and beta-thalassemia, known as ARU-1801, has been named an orphan drug by the U.S. Food and Drug Administration (FDA), a status ... Read more

January 21, 2020January 21, 2020

FT-4202, FORMA’s Experimental SCD Therapy, Fares Well in Phase 1 Trial of Healthy Volunteers

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FT-4202, FORMA Therapeutics’ experimental disease-modifying therapy for sickle cell disease (SCD), shows a favorable safety and pharmacokinetic profile in healthy volunteers, according to data from a Phase 1 clinical ... Read more