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…

Sarepta Acquires Investigational Gene Therapy for LGMD2A

Sarepta Therapeutics has executed an exclusive license agreement for a gene therapy candidate that aims to treat limb-girdle muscular dystrophy type 2A (LGMD2A), the company announced. The investigational therapy, called calpain 3 or CAPN-3, was developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital, in Columbus,…

FDA OKs Trial of Investigational DM1 Therapy

The U.S. Food and Drug Administration (FDA) has cleared Avidity Biosciences to start a Phase 1/2 clinical trial testing the company’s investigational therapy AOC 1001 in adults with myotonic dystrophy type 1 (DM1). “The FDA clearance to proceed with our Phase 1/2 clinical trial for AOC 1001…

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…

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…

Free DMD Newborn Screening Now Offered at Boston Hospital

CureDuchenne has teamed up with Brigham and Women’s Hospital, in Boston, to launch the nation’s first free newborn screening initiative for Duchenne muscular dystrophy (DMD). The partnership establishes Brigham and Women’s as the only U.S. birth hospital to offer parents the choice of screening their new baby for…

Rare Myopathy Affects Women, Too, Advocate Emphasizes

After being diagnosed as a carrier of x-linked myotubular myopathy (XLMTM) and losing her infant twin boys to the disease, Ashley Walker was driven into an episode of alcoholism. What she didn’t know then was that her liver was affected severely by the disease, which eventually resulted in an…

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…