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…
News
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…
Medications that activate the protein soluble guanylyl cyclase (sGC) may have promise in the treatment of sickle cell disease (SCD), a new study in cells and animal models indicates. The study, “Beneficial Effects of Soluble Guanylyl Cyclase Stimulation and Activation in Sickle Cell Disease Are…
It took one year for Dona Krystosek to get a diagnosis for her son, Levi, after he was born. The family received three misdiagnoses of fatal diseases until they found out Levi has Jansen’s metaphyseal chondrodysplasia — an extremely rare form of dwarfism.  “The hardest…
Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of sickle cell disease (SCD). Given to candidate therapies with promising clinical data for diseases of high unmet…
Recent Posts
- Newborn test screens for sickle cell disease, SMA, and SCID at once
- Managing acute chest syndrome and sickle cell disease
- A single word from mother continues to speak volumes about compassion
- How workplace adjustments allow us to contribute on an equal footing
- New grants fund research into bone pain, damage in sickle cell disease