News

January 13, 2021January 13, 2021

Roctavian, Hem A Gene Therapy, Sustains Drop in Bleeding Rates, Phase 3 Data Show

News

Roctavian, an investigational gene therapy, significantly reduced bleeding rates and the need for other treatments in men with severe hemophilia A over at least one year, top-line data from a ... Read more

January 12, 2021January 12, 2021

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

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The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD ... Read more

January 11, 2021January 14, 2021

Parents, Lacking Information, Wary of Gene Therapies, UK Survey Finds

News

Parents of children with hemophilia in the U.K. are generally aware of gene therapy, but many lack both the knowledge necessary for an informed decision and confidence regarding this form ... Read more

January 8, 2021January 8, 2021

Hemlibra Lowered Number of Hemophilia A Bleeds Over Nearly 6 Years

News

When given as a preventive treatment for nearly six years, Hemlibra (emicizumab) safely and effectively lowered the number of bleeding episodes in patients with severe hemophilia A, according to ... Read more

January 6, 2021January 8, 2021

Non-viral Gene Therapy Shows Promise in Hemophilia A Animal Models

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Note: This story was updated Jan. 8, 2021, to clarify that the second set of experiments in mice and non-human primates used a research construct, not the gene therapy. ... Read more

January 4, 2021January 4, 2021

uniQure’s Hemophilia B Gene Therapy Program Placed on Hold

News

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on uniQure’s gene therapy program for hemophilia B, which includes AMT-061 (etranacogene dezaparvovec), due to a possibly related ... Read more

December 21, 2020December 21, 2020

Cost-Benefit Analysis Set for Advate, Hemophilia A Treatment

News

Prime Therapeutics and Takeda will work together to establish the value of Advate, relative to its costs and benefits, as a treatment for hemophilia A. According to Prime, work comparing ... Read more

December 18, 2020December 18, 2020

Funding Set for TRM-201 Trial for Joint Disease in Hemophilia

News

Funding has been set for the RESET-HA Phase 3 clinical trial evaluating Tremeau Pharmaceuticals‘ TRM-201 (rofecoxib) in people with hemophilic arthropathy — a painful and degenerative disease caused by ... Read more

December 16, 2020December 16, 2020

FLT180a Gene Therapy Can Prevent Bleeds in Hemophilia B Patients for Up to 3 Years, Data Show

News

A single dose of the experimental gene therapy FLT180a can safely maintain the activity levels of factor IX (FIX) within a normal range for nearly three years, effectively preventing ... Read more