News

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…

The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to CTX001, an investigational gene-editing cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The announcement was made by CRISPR Therapeutics and Vertex…

An advisory committee of the Sickle Cell Disease Association of America is recommending that people with sickle cell disease (SCD) be considered a “vulnerable” population during the reopening of the U.S. economy, and remain at home as much as possible. Patients should also continue taking their medications, and adopt…

The U.S. National Institutes of Health (NIH) has awarded researchers at Rice University a four-year, $2.45-million grant to support the development of a CRISPR/Cas9-based gene editing treatment for sickle cell disease (SCD). Funding preclinical research, the R01 grant seeks to advance a way to modify…

APCER Life Sciences has released a report confirming the safety profile of Endari (L-glutamine), according to Emmaus Life Sciences, which developed the therapy. Endari is an oral therapy for sickle cell disease (SCD) approved by the U.S. Food…

FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…

Fera Pharmaceuticals submitted an application to the U.S. Food and Drug Administration (FDA) requesting orphan drug status for naproxcinod, a potential treatment for for sickle cell disease (SCD). Orphan drug designation is given to experimental treatments for rare diseases (those that affect…

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…

A team at the Case Western Reserve University has been awarded up to $3.7 million from the National Heart, Lung, and Blood Institute (NHLBI) to conduct early studies of emerging gene therapies for sickle cell disease (SCD). Led by Umut Gurkan,…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…