News

NORD Rare Disease Summit, Online Oct. 18-19, Open for Registration

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…

New R&D Center Will Focus on Cell-based Therapies

Biopharmaceutical company Brooklyn ImmunoTherapeutics has launched a new center focused on research and development (R&D) of gene editing and cell-based therapies to treat cancer and rare blood disorders, including sickle cell disease. “Our new R&D facility will provide us with the ability to advance our research and development…

Advocates Lobby US Congress During Virtual Rare Disease Week

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…

Endari Now More Accessible to Texas Medicaid Patients

Endari (L-glutamine), an approved oral treatment for sickle cell disease (SCD), has now been added to the Preferred Drug List in Texas. The move will make Endari more accessible to people in the state who are insured through Medicaid, the government program that provides health insurance to low-income…

Hemanext Teams Up with SC101 to Promote Sickle Cell Awareness

Medical technology company Hemanext is partnering with the Silicon Valley-based nonprofit Sickle Cell 101 (SC101) to promote sickle cell disease education and awareness. The new partners will sponsor local, national, and global efforts to bring attention to the unmet needs of the more than 20 million people…

US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…

UCSF Awarded Nearly $8.4M for CRISPR_SCD001 Gene Editing Trial

The University of California, San Francisco (UCSF) has been awarded $8.39 million to fund a Phase 1/2 clinical trial of CRISPR_SCD001, the first non-viral and CRISPR-Cas9-based gene editing therapy for sickle cell disease (SCD). Researchers will use the gene-editing technology to attempt to correct a genetic mutation in the blood…

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…