Hemophilia Federation of America is a national nonprofit organization that assists, educates, and advocates for the bleeding disorders community.
Genentech, a member of the Roche Group announced today positive data from the primary analysis of the Phase III HAVEN 1 study in adults and adolescents and interim analysis of the Phase III HAVEN 2 study in children evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) for the treatment of hemophilia A with inhibitors to factor VIII. […]
On April 16, 2017 Genentech announced interim results from the Phase III HAVEN 2 study evaluating emicizumab prophylaxis in children less than 12 years of age with hemophilia A and inhibitors to factor VIII. According to this Genentech’s press releases, “interim analysis after a median of 12 weeks of treatment, emicizumab prophylaxis showed a clinically meaningful reduction in the […]
On April 11, Catalyst Biosciences, a clinical-stage biopharmaceutical company focused on developing novel medicines to address hematology indications, reached a key milestone towards starting human trials with completion of the CB 2679d/ISU304 toxicology studies. Their press release can be read in full here.
Spark Therapeutics issued a press release on updated preliminary data from 10 infused participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for hemophilia B. The data is being presented at Hemostastis & Thrombosis Research Society (HTRS) this week. The press release can be read in full here.
Note: The following is an edited version of a press release from Spark Therapeutics. Read the press release from Spark Therapeutics in it’s entirety here. Spark Therapeutics has announced that its Phase 1/2 trial for the investigation of gene therapy for hemophilia A (factor VIII deficiency) has begun. This trial will initially be enrolling participants at the Children’s Hospital […]
Note: The below is an edited version of an article originally published on Globe Newswire. The original release can be read in its entirety here. uniQure N.V. yesterday announced updated clinical data from its ongoing Phase I/II trial of AMT-060, its proprietary, investigational gene therapy, in patients with severe hemophilia B. The updated data show that […]
Note: The below is an edited version of a press release by Spark Therapeutics. The original release can be read in it’s entirety here. PHILADELPHIA, June 13, 2016 – Spark Therapeutics announced today updated results of the first cohort from the ongoing Phase 1/2 clinical trial of SPK-9001, the lead investigational candidate in its SPK-FIX program, which is being studied for the […]
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