The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is…
News
The European Commission and the U.S. Food and Drug Administration have granted orphan drug status to CSL889, an investigational therapy for the treatment of acute vaso-occlusive crises (VOCs) in sickle cell disease (SCD), CSL Behring, the medicine’s maker, announced in a press…
The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent vaso-occlusive crises (VOCs) in people, 16 and older, with sickle cell disease (SCD). The approval covers the use of Adakveo in combination with hydroxyurea (also known as hydroxycarbamide) — another approved therapy…
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…
FTX-6058, an investigational oral treatment being developed by Fulcrum Therapeutics for sickle cell disease (SCD) and beta-thalassemia, is entering a Phase 1 clinical trial in healthy volunteers, the company announced. The trial (NCT04586985), not yet enrolling, aims to recruit 88 adults at its single Altasciences Clinical Kansas site. The…
Siklos (hydroxyurea) — indicated for the treatment of children 2 and older with sickle cell disease — is now available in 100 mg scored tablets, in addition to the 1,000 mg triple-scored tablet form. Scored tablets make measuring medicine more flexible, giving patients greater control in finding…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
Cyclerion Therapeutics has decided to suspend the development of olinciguat, its investigational oral therapy for sickle cell disease (SCD), after the treatment failed to show adequate activity in a Phase 2 clinical trial, the company announced. The STRONG-SCD (NCT03285178), which completed patient enrollment…
The National Institutes of Health (NIH) has awarded a $7.1 million grant to University of Illinois Chicago (UIC) researchers to evaluate the effectiveness of two therapeutic strategies as alternatives to opioids in the management of chronic pain in people with sickle cell disease (SCD). The grant was awarded to co-principal investigators …
A new consortium has been launched by the Critical Path Institute (C-Path) to support the development of safe and effective treatments for people with sickle cell disease. Called the Critical Path for Sickle Cell Disease (CP-SCD), the…
Recent Posts
- What I’ve learned about sickle cell-related hospital admissions
- Why I go into overdrive for Sickle Cell Awareness Month every September
- Balancing my education goals with my sickle cell needs
- Advocates rally across US for Sickle Cell Awareness Month
- Transforming how others see life with sickle cell disease