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,”…
News
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…
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to GlycoMimetics’ investigational therapy rivipansel (GMI-1070) for the treatment of sickle cell disease (SCD). This designation is given to experimental treatments that have the potential to provide clinically meaningful benefits to patients, primarily…
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…
FTX-6058, an investigational treatment for sickle cell disease (SCD) and beta-thalassemia being developed by Fulcrum Therapeutics, showed efficacy at raising the levels of fetal hemoglobin in cellular and animal models of the diseases, according to new preclinical data announced by the company. Fulcrum also stated…
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