As the season of love and romance approaches, my husband, Jared, and I are already brainstorming ideas for possible February dates. We’re no strangers to unconventional coupledom. A trip to a park or a museum trumps a stroll at the mall; a night filled with laughter while watching a comedy…
Hemgenix (etranacogene dezaparvovec), the first and only one-time gene therapy approved for hemophilia B, has been approved in Switzerland, CSL Behring, the company marketing the therapy globally, announced. “The approval of Hemgenix in Switzerland marks an important milestone for patients, and we look forward to collaborating with…
Almost three years of treatment with Jivi (damoctocog alfa pegol) reduced the annual bleeding rate among a large, multinational group of previously treated hemophilia A patients, a real-world study reports. The number of patients without joint bleeds increased during the study and…
The new year ushered in an unexpected surprise: the near demise of my six-year-old laptop. I lifted the rose gold aluminum top expecting business as usual only to find a huge black line running from the top of my screen to the bottom. To my horror, I also discovered the…
Giroctocogene fitelparvovec, formerly known as SB-525 or PF-07055480, is a gene therapy being developed to reduce the risk of bleeding episodes in people with hemophilia A.
Albumin fusion is a molecular technology that has been used to create replacement therapies for hemophilia with an extended half-life — treatments that are more long-lasting in the body and can therefore be administered less frequently.
CRISPR/Cas9 is a gene-editing technology that can be used to alter the genetic sequence of a specific gene inside a cell. Several preclinical studies have indicated that the molecular tools in CRISPR/Cas9 might be used to treat or even cure hemophilia.
The first patient has been dosed in China in a registrational clinical trial of BBM-H803, a one-time investigational gene therapy that Belief BioMed is developing to prevent bleeding in hemophilia A. The Phase 1/2/3 clinical trial (CTR20233400) is testing how safe and well tolerated BBM-H803 is as a…
Although hemophilia, a bleeding disorder that interferes with blood clotting, is more common in men, women can have it, too.
Health Canada has approved fidanacogene elaparvovec, formerly known as SPK-9001 and now marketed under the brand name Beqvez, for adults with moderately severe to severe hemophilia B. Developed by Pfizer, the gene therapy is under review by regulatory agencies in the U.S. and Europe. “Pfizer has…
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