Diagnosis & Disease Information

blood cells

Valoctocogene Roxaparvovec Offers Stable and Durable Bleed Control in Hemophilia A

BioMarin Pharmaceuticals released positive results from its global 3-year phase 3 study of valoctocogene roxaparvovec (Roctavian™) for patients with hemophilia A. With 134 participants, GENEr8-1 is the largest phase 3 study on gene therapy for hemophilia to date. “We continue to learn more about the durability, safety and efficacy of valoctocogene roxaparvovec,” said Steven Pipe,…

study results

Top-Line Results Released From Hemophilia B Study of Fidanacogene Elaparvovec

Pfizer has announced positive results from its phase 3 BENAGENE-2 study assessing the noninferiority and superiority of fidanacogene elaparvovec gene therapy for moderately severe to severe hemophilia B. The results, announced via news release, showed the treatment met the primary superiority endpoint in terms of annualized bleeding rate (ABR) of total bleeds after infusion of…

Rare Care podcast logo

An Interview With PJ Brooks, PhD, on the Bespoke Gene Therapy Consortium

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Philip J. Brooks, PhD, acting director of the Division of Rare Diseases Research Innovation at the National Institutes of Health’s National Center for Advancing Translational Sciences.

LAL-D experimental therapies

Hematopoietic Stem Cell Gene Therapy May Be Beneficial in PD Management

Lentiviral vector-mediated hematopoietic stem and progenitor cell (HSPC) gene therapy may lead to increased acid alpha-glucosidase (GAA) levels in animal models, demonstrating its therapeutic potential in Pompe disease (PD), according to a recently published study in Methods and Clinical Development.  Currently, enzyme replacement therapy (ERT) constitutes the standard of care for patients with PD, which has…

Adeno-associated viruses

Gene Therapy Could Provide Decades-Long Relief From FIX Prophylaxis for Hemophilia B

Researchers reported that patients with hemophilia B who received a single administration of etranacogene dezaparvovec would likely have enough factor IX (FIX) levels to remain free from the need for prophylactic FIX replacement products for more than 2 decades, according to a study published in Current Medical Research and Opinion.  “Gene therapy offers the potential…

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