JCR Pharmaceuticals and Sumitomo Dainippon Pharma have teamed up to market and commercialize in Japan agalsidase beta BS, a biosimilar of Fabrazyme, an already approved treatment designed to replace a critical enzyme missing in people with Fabry disease. According to a company press release, “This…
The next patient to receive FLT190, an investigational gene therapy for Fabry disease, in the ongoing Phase 1/2 MARVEL-1 trial will be given a second, higher dose of therapy, accelerating an earlier trial timeline. This person was originally intended to be included in the first, lowest dose group of…
Healthcare providers involved in diagnosing and treating rare diseases believe that increased physician education and collaboration with specialized facilities will have the greatest positive impact on treating these conditions over the next five years, according to results from a 2021 survey. Definitive Healthcare, a healthcare commercial intelligence company, conducted…
Updated findings in an ongoing Phase 1/2 clinical trial of Sangamo Therapeutics‘ gene therapy ST-920 (isaralgagene civaparvovec) shows the treatment continues to be well tolerated and effective in adults with Fabry disease. As of the Nov. 19 data cutoff date, all five treated patients enrolled showed normalized levels of…
Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…
Experimental gene therapy 4D-310 continues to be generally safe and has effectively increased enzyme alpha-galactosidase (Gal A) blood levels in the first three patients enrolled in 4D Molecular Therapeutics’s clinical trial. These results were announced in the presentation “An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in…
Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million people worldwide living with a rare disease; for many of them, access to diagnosis, care, and treatments can be challenging. Accessing better care depends on…
Protalix BioTherapeutics and its partner Chiesi Global Rare Diseases have applied to the European Medicines Agency (EMA) for the approval of PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy, to treat adults with Fabry disease. This approval request, in the form of a marketing authorization application, has been validated by the EMA and now…
Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like to live with a rare disease, including conversations about advocacy, mental health, survivor’s guilt, treatment of minority patients, and more. The Monday event, “A…
A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…
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