Hemophilia Federation of America is a national nonprofit organization that assists, educates and advocates for the bleeding disorders community.
The following is an excerpt from a press release from Sangamo Therapeutics. Read the press release in its entirety here. Sangamo Therapeutics Inc., a genomic medicine company, and Pfizer Inc. announced interim data from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A. Data indicate that SB-525 was generally well-tolerated […]
The following is an excerpt from a press release from Spark Therapeutics. Read the press release in its entirety here. Spark Therapeutics , a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, announced today that it has entered into a definitive merger agreement for Roche to fully acquire Spark […]
The following is a press release from Roche. Read the press release in its entirety here. Roche announced it has joined the World Federation of Hemophilia Humanitarian Aid Program, a landmark initiative leading the effort to change the lack of access to care and treatment for people with inherited bleeding disorders in developing countries. Together […]
The following is an excerpt from a press release. Read the press release in its entirety here. Denmark-based Novo Nordisk received approval from the U.S. Food and Drug Administration for a new treatment for hemophilia A. However, the newly approved medication will not be available in the United States until 2020. Novo Nordisk said the […]
The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced updated clinical data in patients treated in the company’s ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a […]
The following is an excerpt from a press release from Roche. Read it in its entirety here. Roche today announced that it has joined the World Federation of Hemophilia Humanitarian Aid Program, a landmark initiative leading the effort to change the lack of access to care and treatment for people with inherited bleeding disorders in […]
The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced that it treated the first patient in its HOPE-B pivotal trial of AMT-061, an investigational AAV5-based gene therapy incorporating the […]
The following is a press release from Takeda, formerly Shire. Find the original press release here. Takeda Pharmaceutical Company Limited announced the completion of its acquisition of Shire, becoming a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan. Takeda now has an attractive, expanded geographic footprint and leading position in Japan and the U.S., bringing […]
The following is an excerpt from a press release from Save One Life Inc. Read the press release in its entirety here. Save One Life Inc. announces a new executive director. Save One Life is an international nonprofit that assists people with hemophilia in developing countries. Christopher G. Bombardier, famous for being the first person […]
The following is an excerpt from the Plasma Protein Therapeutics Association. Read the full press release here. The Board of Directors of the Plasma Protein Therapeutics Association announced Amy Efantis will be its next President and Chief Executive Officer effective Jan. 16, 2019. Having served most recently as Vice President, Global Public Policy & […]
The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech, a member of the Roche Group announced data from the primary analysis of the Phase III HAVEN 2 study evaluating Hemlibra® prophylaxis in children younger than 12 years of age with hemophilia A with factor […]
The following is an excerpt from a press release from Sangamo. Read the press release in its entirety here. Hemophilia B is a rare, genetic bleeding disorder caused by a lack of the factor IX (FIX) protein, which is necessary for normal blood clotting. Current hemophilia B treatments typically require frequent intravenous infusions of clotting […]
The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech, a member of the Roche Group, announced data from the primary analysis of the Phase III HAVEN 2 study evaluating Hemlibra® prophylaxis in children younger than 12 years of age with hemophilia A with factor VIII […]
The following is an excerpt from a press release from Bioverativ. Read the press release in its entirety here. Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, presented new data from the EXTEN-A Phase 1/2a trial of BIVV001 showing that a single 65 IU/kg dose of BIVV001 extended […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated results from its ongoing Phase I/II trial of AMT-060, and provided an update on AMT-061, the company’s next-generation gene therapy candidate […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure announces the initial topline data from our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B. All patients achieved and sustained therapeutic […]
The following is a press release from uniQure. uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, announced the expansion of its research pipeline with novel AAV gene therapy approaches to treating Hemophilia A, Fabry disease and Spinocerebellar Ataxia Type 3 at the Company’s Research & Development Day held this […]
The following is an excerpt from a press release from uniQure. uniQure announced that they have treated the first patient in our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B (see attached press release). “As a one-time administered therapy, AMT-061 has the […]
The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech has announced new data that will be presented at this year’s annual American Society of Hematology meeting held in San Diego from Dec. 1-4. Ten Genentech medicines will be featured in more than 70 abstracts, including […]
The following is an excerpt from a press release from Genentech. Read the entire press release here. Genentech, a member of the Roche Group, announced that the U.S. Food and Drug Administration has approved Hemlibra®(emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, […]
El siguiente es un comunicado de prensa proporcionado por Genentech. LA FDA APRUEBA HEMLIBRA® DE GENENTECH (EMICIZUMAB-KXWH) PARA LA HEMOFILIA A SIN INHIBIDORES DE FACTOR VIII – El primer medicamento que reduce considerablemente la hemorragia tratada en comparación con la previa profilaxis de factor VIII basada en una comparación intrapaciente – – Único medicamento que puede […]
Genentech has announced a new web portal for patients and caregivers intended to provide timely and accurate information on targeted serious adverse events of interest for HEMLIBRA. Visit the safety website: http://www.emipatientinfo.com/. Earlier this year, Genentech launched a similar site specifically for health care providers. In announcing the launch, Genentech stated “This website is meant to […]
The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech, a member of the Roche Group, announced that pivotal data from the Phase III HAVEN 3 study, which evaluated HEMLIBRA® prophylaxis administered every week or every two weeks in adults and adolescents aged 12 years or […]
The following is an excerpt from a press release from Bayer. Read the press release in its entirety here. Bayer announced that the U.S. Food and Drug Administration has approved Jivi® for the routine prophylactic treatment of hemophilia A in previously-treated adults and adolescents 12 years of age or older. The initial recommended prophylactic regimen […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced it has treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy […]
On Aug. 7, 2018, the U.S. District Court in Delaware ruled against Shire in its request for a preliminary injunction against Roche AG from creating, transporting and marketing its recently-approved drug Hemlibra in the U.S., which received FDA approval to treat people with hemophilia A with inhibitors in 2017. This ruling means there are no […]
The following is an excerpt from a press release from Spark Therapeutics. Read the press release in its entirety here. Investigational SPK-8011 moving to Phase 3 As of the July 13 data cutoff, 12 participants in the Phase 1/2 trial have received a single administration of investigational SPK-8011, including two at a dose of 5×1011 […]
The following is a summary of a press release from Catalyst Biosciences by Dr. Robert Sidonio, a trusted medical advisor. Read the full press release here. On June 18, Catalyst Biosciences provided an update of the progress from its Phase ½ trial investigating the use of FIX variant product, called CB 2679d/ISU304, which is a […]
The following is an excerpt from a press release from Genentech. Read the entire press release here. Genentech, a member of the Roche Group, announced the U.S. Food and Drug Administration has accepted the company’s supplemental Biologics License Application and granted Priority Review for HEMLIBRA  for adults and children with hemophilia A without factor VIII […]
The following is an excerpt from a press release from BioMarin. Read the entire press release 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 updated results were presented during […]
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