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BioMarin Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. The data […]

Industry News

Spark Therapeutics and Pfizer Announce Longer-term Preliminary Data Showing Consistent and Sustained Factor IX Levels in Hemophilia B

Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. Spark Therapeutics and Pfizer Inc. announced on Dec. 11, 2017, with a cumulative follow-up of more than 13 patient years of observation, all 11 participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for […]

Industry News

Spark Therapeutics Presents Preliminary Data on Investigational SPK-8011 Phase 1/2 Dose-escalation Clinical Trial in Hemophilia A

Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. Spark Therapeutics announced on Dec. 11, 2017, it has dosed seven participants in the Phase 1/2 clinical trial of SPK-8011 in hemophilia A. The first four participants, who have been followed at least 12 weeks post […]

Industry News

Bioverativ Announces Results of Eloctate Study Showing High Bleed Protection and Target Joint Resolution

Note: The following is edited from a press release from Bioverativ. Read the full press release in its entirety here. Bioverativ Inc. and Swedish Orphan Biovitrum AB announced the results of a new, post-hoc, longitudinal analysis of the pivotal Phase 3 A-LONG study and ASPIRE long-term extension study, showing that weekly prophylactic dosing with its […]

Industry News

Bioverativ Announces Findings From Novel Imaging Study Using Alprolix

Note: The following is edited from a press release from Bioverativ. Read the full press release in its entirety here. Bioverativ Inc., a global biopharmaceutical company dedicated to transforming the lives of people with rare blood disorders, today announced findings from a novel imaging study investigating extravascular distribution of factor IX therapies, including its leading extended […]

Industry News

FDA Lifts Clinical Hold on Fitusiran

Note: The following is edited from a press release from Alnylam Pharmaceuticals. Read the full press release in its entirety here. Alnylam Pharmaceuticals Inc., a RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced Dec. 15, 2017, the U.S. Food and Drug Administration (FDA) has lifted the hold on clinical studies with fitusiran, including […]

Industry News

Genentech Presents New Data on Hemlibra at ASH 2017

Note: The following is edited from a press release from Genentech. Read the full press release in its entirety here. Genentech, a member of the Roche Group, announced on Dec. 9, 2017, new data from the ongoing HEMLIBRA® (emicizumab-kxwh) clinical development program were presented at the 59th American Society of Hematology (ASH) Annual Meeting. These data […]

Industry News

Shire Files Preliminary Injunction Against Genentech/Roche

Note: The following is edited from a press release from Shire. Read the full press release in its entirety here. On December 14, 2017, Shire filed a motion for preliminary injunction against Roche subsidiaries Genentech Inc. and Chugai Pharmaceutical Co., Ltd., as part of an ongoing U.S. patent infringement lawsuit surrounding Genentech/Roche’s emicizumab, currently being marketed as […]

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Infusing Love: Through the Smoke

It’s been two months since wildfires ripped through Northern California leaving a path of destruction in Sonoma and Napa counties like I’ve never seen before and leaving a scar on all of our hearts. I got to see firsthand the importance of community and was proud to see our bleeding disorder community come together to […]

Dear Addy: Take Action

Dear Addy, What is the best way for me to contact my legislators when policy issues affecting the bleeding disorders community arise? Signed, Alert Advocate Dear Advocate, HFA’s Take Action Center has all you need to contact your legislators, including talking points on issues affecting the bleeding disorders community. Watch this short video on using the […]

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