Dosing Differences Opined for Costly Hemophilia Option Dosing Differences Opined for Costly Hemophilia Option
Achieving the goal of zero bleeds as patients pursue active lives will require divergent dosing, according to PROPEL trial investigators who evaluated whether more exposure to FVIII improves outcomes.Medscape Medical News (Source: Medscape Hematology-Oncology Headlines)
Source: Medscape Hematology-Oncology Headlines - July 12, 2019 Category: Cancer & Oncology Tags: Hematology-Oncology News Source Type: news

Local biotechs UniQure, Casebia step up race for new hemophilia drugs
The race for new, long-lasting treatments for the two most common forms of hemophilia heated up this week as two local gene therapy companies presented trial data for drugs in development alongside Big Pharma players. Lexington biotech UniQure N.V. (Nasdaq: QURE) — which has been the subject of M&A rumors in recent weeks, per Bloomberg — presented new data earlier this week on its treatment for hemophilia B, a form of the rare, genetic blood disease which affects the body’s ability to form… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - July 11, 2019 Category: Biotechnology Authors: Allison DeAngelis Source Type: news

BioMarin Appoints Pharmaceutical Veteran and Former J and J Executive, Liz McKee Anderson, to Board of Directors
SAN RAFAEL, Calif., July 9, 2019 -- (Healthcare Sales & Marketing Network) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN), a global leader in providing therapies for rare genetic diseases, today announced the appointment of pharmaceutical veteran and forme... Biopharmaceuticals, Personnel BioMarin Pharmaceutical, valoctocogene roxaparvovec, hemophilia A, gene therapy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - July 9, 2019 Category: Pharmaceuticals Source Type: news

Roche presents a broad range of data for Hemlibra demonstrating continued benefits for people with haemophilia A at the ISTH 2019 Congress
Roche today announced new data for Hemlibra ® (emicizumab) across multiple pivotal studies in people with haemophilia A with and without factor VIII inhibitors at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress on 6-10 July in Melbourne, Australia. (Source: Roche Investor Update)
Source: Roche Investor Update - July 9, 2019 Category: Pharmaceuticals Source Type: news

Roche presents a broad range of data for Hemlibra demonstrating continued benefits for people with haemophilia A at the ISTH 2019 Congress
Roche today announced new data for Hemlibra ® (emicizumab) across multiple pivotal studies in people with haemophilia A with and without factor VIII inhibitors at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress on 6-10 July in Melbourne, Australia. (Source: Roche Media News)
Source: Roche Media News - July 9, 2019 Category: Pharmaceuticals Source Type: news

Contaminated blood scandal: Victims reveal 'coalition of secrecy'
Haemophiliac Alistair Bennett contracted HIV/Aids from a contaminated blood product. He died aged 22. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - July 2, 2019 Category: Consumer Health News Source Type: news

New plasma donation center takes space on Gadsden Highway
A new plasma donation center is set to open on July 10, at 222 Gadsden Highway in Birmingham.   OctaPharma is a Lachen, Switzerland-based company that allows people to donate plasma for medical production.  “OctaPharma converts donated plasma into plasma protein products used in more than 100 countries to treat life-threatening congenital and acquired diseases such as hemophilia A and B, immune deficiency syndromes, Rh disease in newborns and burn injuries,” Dr. David J. Aarons, medical director… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - June 18, 2019 Category: Biotechnology Authors: Tyler Patchen Source Type: news

BioMarin says data shows hemophilia gene therapy effects could wane
BioMarin Pharmaceutical Inc said on Tuesday early trial data for its gene therapy for hemophilia A suggested the one-time infusion's effect on some patients' bleeding disorders would last eight years. (Source: Reuters: Health)
Source: Reuters: Health - May 28, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

A hemophilia patient hero at zero, BioMarin to seek FDA OK for gene therapy. But will the effect last?
A potential one-shot-and-you're-done hemophilia A treatment from BioMarin Pharmaceutical Inc. will target regulatory approval in the United States and Europe after showing dramatic decreases in bleeding during clinical trials. But in a competitive market to find the next big and expensive treatment to help — or even cure — hemophilia A patients, questions linger around how long the treatment sticks with patients. BioMarin (NASDAQ: BMRN) apparently didn't allay those concerns Tuesday: The San… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - May 28, 2019 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

A hemophilia patient hero at zero, BioMarin to seek FDA OK for gene therapy. But will the effect last?
A potential one-shot-and-you're-done hemophilia A treatment from BioMarin Pharmaceutical Inc. will target regulatory approval in the United States and Europe after showing dramatic decreases in bleeding during clinical trials. But in a competitive market to find the next big and expensive treatment to help — or even cure — hemophilia A patients, questions linger around how long the treatment sticks with patients. BioMarin (NASDAQ: BMRN) apparently didn't allay those concerns Tuesday: The San… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - May 28, 2019 Category: Biotechnology Authors: Ron Leuty Source Type: news

BioMarin Announces that Phase 3 Cohort of Valoctocogene Roxaparvovec, Gene Therapy Study in Severe Hemophilia A Met Pre-Specified Criteria for Regulatory Submissions in the U.S. and Europe
Timing of Regulatory Submissions to Be Determined in 3Q 2019 Conference Call and Webcast to be Held Tuesday, May 28, 2019 at 8:00 AM Eastern SAN RAFAEL, Calif., May 28, 2019 -- (Healthcare Sales & Marketing Network) -- BioMarin Pharmaceutical Inc. (... Biopharmaceuticals BioMarin Pharmaceutical, valoctocogene roxaparvovec, hemophilia A, gene therapy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - May 28, 2019 Category: Pharmaceuticals Source Type: news

Researchers ready B cells for novel cell therapy
(Seattle Children's) Scientists at Seattle Children's Research Institute are paving the way to use gene-edited B cells -- a type of white blood cell in the immune system -- to treat a wide range of potential diseases that affect children, including hemophilia and other protein deficiency disorders, autoimmune diseases, and infectious diseases. If successful, their research would open the door to offering this experimental cell therapy as the first-of-its-kind in clinical trials at Seattle Children's in as soon as five years. (Source: EurekAlert! - Infectious and Emerging Diseases)
Source: EurekAlert! - Infectious and Emerging Diseases - May 2, 2019 Category: Infectious Diseases Source Type: news

PHARMAC funds longer-acting treatments for haemophilia
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - April 30, 2019 Category: Drugs & Pharmacology Source Type: news

Enzyre Appoints Dirk Pollet as Chief Executive Officer
Awarded €2M grant and completes seed financing to develop high-precision point-of-care Hemophilia Biochip device NIJMEGEN, Netherlands, April 30, 2019 -- (Healthcare Sales & Marketing Network) -- Enzyre, which is developing breakthrough ambulant diagno... Diagnostics, Personnel Enzyre , Hemophilia Biochip (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - April 30, 2019 Category: Pharmaceuticals Source Type: news

St. Jude prepares to launch global hemophilia gene therapy trial
St. Jude is trying to establish a gene therapy program to bring next-level treatment options to patients in under-resourced countries. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - April 23, 2019 Category: Biotechnology Authors: Jason Bolton Source Type: news