CHOP-led research study identifies key target in treatment-resistant hemophilia A
(Children's Hospital of Philadelphia) Researchers at Children's Hospital of Philadelphia (CHOP) have identified a key target that may be responsible for treatment failure in about 30% of patients with hemophilia A. The target, known as B cell activating factor (BAFF), appears to promote antibodies against and inhibitors of the missing blood clotting factor that is given to these patients to control their bleeding episodes. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 15, 2021 Category: International Medicine & Public Health Source Type: news

BioMarin Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to Valoctocogene Roxaparvovec, Investigational Gene Therapy for Hemophilia A
RMAT Designation Granted by FDA During Bleeding Disorders Awareness Month SAN RAFAEL, Calif., March 8, 2021 -- (Healthcare Sales & Marketing Network) -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administrat... Biopharmaceuticals, FDA BioMarin, Valoctocogene Roxaparvovec, Hemophilia (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - March 8, 2021 Category: Pharmaceuticals Source Type: news

Fidelis Specialty Pharmacy Selects Citus Health to Enable Real-Time...
Innovative specialty pharmacy provider for hemophiliac and bleeding disorder patients to implement Citus Health’s HIPAA-compliant digital health solution suite to improve patient and staff...(PRWeb March 04, 2021)Read the full story at https://www.prweb.com/releases/fidelis_specialty_pharmacy_selects_citus_health_to_enable_real_time_virtual_patient_communications_and_faster_collection_of_key_patient_data/prweb17770242.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - March 4, 2021 Category: Pharmaceuticals Source Type: news

Mission 2030: Bayer ’s quest for climate neutrality
One hundred times per week. That ’s how often specially trained Bayer team members used to clean certain stainless-steel vessels as part of a six-month-long drug manufacturing process. The medicine in question is a biotech treatment for people with a rare blood-clotting disorder. The vessels held buffering agents, solutions that are used in the purification process of making Factor VIII, the protein missing in people living with hemophilia A. Cleaning each vessel required significant amounts of… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - February 26, 2021 Category: Health Management Authors: Lauren Lawley Head Source Type: news

Sudden death of a young hemophiliac by low-velocity blunt knee trauma in bullock cart run-over fatality - Chauhan M, Behera C, Rustagi A.
A young bullock cart driver was pushing bulls hard in stunt and frolic. The cart sped up and he lost control and toppled in front of the iron wheel, which ran over his lower limb around the knee. Concomitant hemophilia further complicated the popliteal art... (Source: SafetyLit)
Source: SafetyLit - February 17, 2021 Category: International Medicine & Public Health Tags: Age: Infants and Children Source Type: news

Roche reports solid results in 2020
Commenting on the Group ’s results, Roche CEO Severin Schwan said: “Roche continues to make important contributions to fighting the COVID-19 pandemic. We developed in record time a comprehensive portfolio of diagnostic solutions and entered new partnerships to develop and produce effective COVID-19 medicines. The deman d for our new medicines which benefit people living with serious conditions, such as cancer, multiple sclerosis, haemophilia and spinal muscular atrophy, remains high. Based on our rejuvenated portfolio and the significant progress made in developing our product pipeline, Roche is strongly positi...
Source: Roche Media News - February 4, 2021 Category: Pharmaceuticals Source Type: news

Roche reports solid results in 2020
Commenting on the Group ’s results, Roche CEO Severin Schwan said: “Roche continues to make important contributions to fighting the COVID-19 pandemic. We developed in record time a comprehensive portfolio of diagnostic solutions and entered new partnerships to develop and produce effective COVID-19 medicines. The deman d for our new medicines which benefit people living with serious conditions, such as cancer, multiple sclerosis, haemophilia and spinal muscular atrophy, remains high. Based on our rejuvenated portfolio and the significant progress made in developing our product pipeline, Roche is strongly positi...
Source: Roche Investor Update - February 4, 2021 Category: Pharmaceuticals Source Type: news

New follow-up phase III data reinforce the long-term benefit of Roche ’s Hemlibra for people with haemophilia A
Basel, 7 December 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced results from a new analysis of pooled, three-year follow-up data of 401 people with haemophilia A from the pivotal HAVEN 1-4 studies, which reinforce the long-term efficacy and safety profile of Hemlibra ® (emicizumab).[1]These data, from adults, adolescents and children with haemophilia A with and without factor VIII inhibitors, were presented at the all-virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 5-8 December 2020.“The long-term benefit of Hemlibra, with a consistent safety profile and durably effect...
Source: Roche Media News - December 7, 2020 Category: Pharmaceuticals Source Type: news

New follow-up phase III data reinforce the long-term benefit of Roche ’s Hemlibra for people with haemophilia A
Basel, 7 December 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced results from a new analysis of pooled, three-year follow-up data of 401 people with haemophilia A from the pivotal HAVEN 1-4 studies, which reinforce the long-term efficacy and safety profile of Hemlibra ® (emicizumab).[1]These data, from adults, adolescents and children with haemophilia A with and without factor VIII inhibitors, were presented at the all-virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 5-8 December 2020.“The long-term benefit of Hemlibra, with a consistent safety profile and durably effect...
Source: Roche Investor Update - December 7, 2020 Category: Pharmaceuticals Source Type: news

On World AIDS Day, Those Who Fought the 1980s Epidemic Find Striking Differences and Tragic Parallels in COVID-19
More than three decades after the World Health Organization (WHO) launched the first World AIDS Day on Dec. 1, 1988, the world’s leading global health organization faces another public health crisis in COVID-19. On this World AIDS Day, those who raised awareness of HIV, the virus that causes AIDS, find devastating similarities and haunting differences in America’s response to both crises. In 1981, scientists recorded the first cases of a rare pneumonia, usually found among immunosuppressed patients, among a group of gay men in Los Angeles, and noticed more cases appearing among gay men in San Francisco and New ...
Source: TIME: Health - December 1, 2020 Category: Consumer Health News Authors: Olivia B. Waxman Tags: Uncategorized COVID-19 feature HIV/AIDS Source Type: news

Roche to present new data across 16 blood disorders at the American Society of Hematology 2020 Annual Meeting
Basel, 5 November 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data for its approved and investigational medicines will be presented at the all-virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition from 5 – 8 December 2020. Eleven Roche medicines will be featured in more than 80 abstracts, including 22 oral presentations. With studies spanning 16 blood disorders, including non-Hodgkin lymphoma (NHL), leukaemia, multiple myeloma (MM) and haemophilia A, these data highlight the strength and breadth o f Roche’s haematology portfolio and pipeline, and commitment to de...
Source: Roche Media News - November 5, 2020 Category: Pharmaceuticals Source Type: news

Roche to present new data across 16 blood disorders at the American Society of Hematology 2020 Annual Meeting
Basel, 5 November 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data for its approved and investigational medicines will be presented at the all-virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition from 5 – 8 December 2020. Eleven Roche medicines will be featured in more than 80 abstracts, including 22 oral presentations. With studies spanning 16 blood disorders, including non-Hodgkin lymphoma (NHL), leukaemia, multiple myeloma (MM) and haemophilia A, these data highlight the strength and breadth o f Roche’s haematology portfolio and pipeline, and commitment to de...
Source: Roche Investor Update - November 5, 2020 Category: Pharmaceuticals Source Type: news

Post-Dental Extraction Bleeding Decreases With Mouth Splints Post-Dental Extraction Bleeding Decreases With Mouth Splints
Dental extractions can cause significant risk of bleeding in patients with hemophilia being treated with factor replacements.Medscape Medical News (Source: Medscape Hematology-Oncology Headlines)
Source: Medscape Hematology-Oncology Headlines - November 4, 2020 Category: Cancer & Oncology Tags: Dental & Oral Health News Source Type: news

PHARMAC funding emicizumab for severe haemophilia A
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - November 1, 2020 Category: Drugs & Pharmacology Source Type: news

26.10.20: Bayer acquires Asklepios BioPharmaceutical to broaden innovation base in cell and gene therapy
Bayer acquires Asklepios BioPharmaceutical to broaden innovation base in cell and gene therapyAcquisition fuels Bayer's cell and gene therapy platform with potential to bring urgently needed treatments to patients across multiple disease areas with high unmet need / AskBio's industry leading AAV-based gene therapy platform already yielding commercial and clinical stage assets with potential of helping larger patient populations / Portfolio includes investigational pre-clinical and clinical stage development candidates for the treatment of neuromuscular, central nervous system, cardiovascular and metabolic diseases such as ...
Source: Bayer IR Newsfeed: Events - October 26, 2020 Category: Pharmaceuticals Source Type: news

Bayer-acquires-Asklepios-BioPharmaceutical-to-broaden-innovation-base-in-cell-and-gene-therapy
Acquisition fuels Bayer’s cell and gene therapy platform with potential to bring urgently needed treatments to patients across multiple disease areas with high unmet need / AskBio's industry leading AAV-based gene therapy platform already yielding commercial and clinical stage assets with potential of helping larger patient populations / Portfolio includes investigational pre-clinical and clinical stage development candidates for the treatment of neuromuscular, central nervous system, cardiovascular and metabolic diseases such as therapeutics for Pompe disease, Parkinson’s disease and congestive heart failure, ...
Source: Bayer Company News - October 26, 2020 Category: Pharmaceuticals Source Type: news

Disregarding Rights of Persons with Disabilities
The focus of the International Day of Persons with Disabilities, which falls on 3 December, is the link between the empowerment of people living with disability, and the 2030 Agenda for Sustainable Development, the UN’s blueprint for a better future for people and the planet. Credit: UN NewsBy Shudarson Subedi and Simone GalimbertiKATHMANDU, Nepal, Oct 15 2020 (IPS) The lack of consistency and a patchy approach undermines the Government of Nepal’s credibility in fulfilling the rights of persons with disabilities. One step forward and several steps back. If we want to describe the current progress being made by...
Source: IPS Inter Press Service - Health - October 15, 2020 Category: International Medicine & Public Health Authors: Shudarson Subedi and Simone Galimberti Tags: Asia-Pacific Development & Aid Featured Global Headlines Health Human Rights IPS UN: Inside the Glasshouse Population TerraViva United Nations Source Type: news

Early Trial Offers New Hope for People With Hemophilia
Title: Early Trial Offers New Hope for People With HemophiliaCategory: Health NewsCreated: 9/9/2020 12:00:00 AMLast Editorial Review: 9/10/2020 12:00:00 AM (Source: MedicineNet Chronic Pain General)
Source: MedicineNet Chronic Pain General - September 10, 2020 Category: Anesthesiology Source Type: news

Early Trial Offers New Hope for People With Hemophilia
WEDNESDAY, Sept. 9, 2020 -- Researchers may have found a way for people with severe hemophilia to take their standard treatment less often, if the results of an early trial pan out. In what experts called a feat of bioengineering, scientists were... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - September 9, 2020 Category: General Medicine Source Type: news

Are new treatments for haemophilia  A value for money?
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - August 31, 2020 Category: Drugs & Pharmacology Source Type: news

Paying for cell and gene therapy - Is the future already here?
The ascent of cell and gene therapies over the past few years has been astonishing. And their rise looks unstoppable: By 2025, the FDA expects it will be reviewing 10 to 20 of these transformative drugs per year.    But as we listen to affordability concerns from payers, providers and patients, we ’ve also had to ask, perhaps a bit provocatively, how we can afford to pay for this boom in future cures?  " Without solutions to help payers manage the cost, some of our members may make the choice to exclude coverage " , stated insurer CVS Health earlier in the year in its position paper on ...
Source: EyeForPharma - August 25, 2020 Category: Pharmaceuticals Authors: Ulrich Neumann Source Type: news

Paying for cell and gene therapy - Is the future already here?
The ascent of cell and gene therapies over the past few years has been astonishing. And their rise looks unstoppable: By 2025, the FDA expects it will be reviewing 10 to 20 of these transformative drugs per year.    But as we listen to affordability concerns from payers, providers and patients, we ’ve also had to ask, perhaps a bit provocatively, how we can afford to pay for this boom in future cures?  " Without solutions to help payers manage the cost, some of our members may make the choice to exclude coverage " , stated insurer CVS Health earlier in the year in its position paper on ...
Source: EyeForPharma - August 25, 2020 Category: Pharmaceuticals Authors: Ulrich Neumann Source Type: news

After surprise rejection of hemophilia gene therapy, BioMarin asks FDA to approve dwarfism drug
A controversial drug to treat the most common form of dwarfism is heading for a federal regulator's decision. BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) of San Rafael said Thursday that it submitted its new drug application for the Food and Drug Administration to approve its drug, called vosoritide, as a once-daily injection for children with achondroplasia. The bone-growth disorder prevents cartilage fr om changing into bone. The application comes a day after the FDA rejected BioMarin's request… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - August 20, 2020 Category: Biotechnology Authors: Ron Leuty Source Type: news

After surprise rejection of hemophilia gene therapy, BioMarin asks FDA to approve dwarfism drug
A controversial drug to treat the most common form of dwarfism is heading for a federal regulator's decision. BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) of San Rafael said Thursday that it submitted its new drug application for the Food and Drug Administration to approve its drug, called vosoritide, as a once-daily injection for children with achondroplasia. The bone-growth disorder prevents cartilage fr om changing into bone. The application comes a day after the FDA rejected BioMarin's request… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - August 20, 2020 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

FDA blocks much-anticipated BioMarin hemophilia gene therapy
BioMarin Pharmaceutical shares plunged by a third after U.S. regulators rejected its potentially game-changing gene therapy for hemophilia A patients (Source: ABC News: Health)
Source: ABC News: Health - August 19, 2020 Category: Consumer Health News Tags: Health Source Type: news

Promising gene therapy against hemophilia rejected by FDA
BioMarin had gone so far as to give its hemophilia A gene therapy a name. Now it will have to deliver more data to the FDA. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - August 19, 2020 Category: Biotechnology Authors: Ron Leuty Source Type: news

BioMarin Receives Complete Response Letter (CRL) from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
SAN RAFAEL, Calif., Aug. 19, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to the Company's Biologics License... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - August 19, 2020 Category: Drugs & Pharmacology Source Type: news

Include Indigenous People in COVID-19 Response
Credit: Nidwan.By Pratima GurungKATHMANDU, Jul 28 2020 (IPS) In Nepal the COVID-19 crisis has been especially hard on indigenous peoples. We had to learn a new vocabulary and use words like quarantine, self-isolation, hand sanitizers and social distancing. We also had to respect rules that did not previously apply to our lives. Indigenous peoples are not used to washing their hands all the time because our culture is so much closer to Mother Earth and because much of the time we don’t have running water. The situation has been even more difficult for indigenous persons with a disability, like me. I cannot keep my so...
Source: IPS Inter Press Service - Health - July 28, 2020 Category: International Medicine & Public Health Authors: Pratima Gurung Tags: Asia-Pacific Headlines Health Human Rights Indigenous Rights Source Type: news

A new cell & gene therapy approach to treat common bleeding disorder
(Wake Forest Baptist Medical Center) WFIRM researchers have developed an optimized cellular platform for delivering Factor 8 to better treat patients with hemophilia A. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 28, 2020 Category: International Medicine & Public Health Source Type: news

Gene Therapy Shows Promise For Hemophilia, But Could Be Most Expensive U.S. Drug Ever
(Source: NPR Health and Science)
Source: NPR Health and Science - July 20, 2020 Category: Consumer Health News Authors: Rob Stein Source Type: news

Roche, Genentech ink real-world data deal with PicnicHealth
The original focus will be on multiple sclerosis but will  extend to include Huntington's disease and hemophilia.   (Source: mobihealthnews)
Source: mobihealthnews - July 16, 2020 Category: Information Technology Source Type: news

Spark Therapeutics Announces Updated Data on SPK-8011 from Phase 1/2 Clinical Trial in Hemophilia A at ISTH 2020 Virtual Congress
Dear Investor, Please find attached a press release by Spark Therapeutics:https://sparktx.com/press_releases/isth2020/ Do not hesitate to contact us for any further questions. With best regards, (Source: Roche Investor Update)
Source: Roche Investor Update - July 13, 2020 Category: Pharmaceuticals Source Type: news

New data from phase IIIb study reinforces safety profile of Roche ’s Hemlibra in people with haemophilia A
Roche today announced results from the second interim analysis of the phase IIIb STASEY study, which reinforce the safety profile of Hemlibra ® (emicizumab) characterised in the phase III HAVEN clinical programme. (Source: Roche Media News)
Source: Roche Media News - July 13, 2020 Category: Pharmaceuticals Source Type: news

New data from phase IIIb study reinforces safety profile of Roche ’s Hemlibra in people with haemophilia A
Basel, 13 July 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced results from the second interim analysis of the phase IIIb STASEY study, which reinforce the safety profile of Hemlibra ® (emicizumab) characterised in the phase III HAVEN clinical programme.1,2,3 In the STASEY study, Hemlibra was effective with no new safety signals identified in adults and adolescents with haemophilia A with factor VIII inhibitors, which was consistent with previous safety observations.1 Further new interim data suggest that people on Hemlibra may be able to undergo certain minor surgeries without additional preventative (prophy...
Source: Roche Investor Update - July 13, 2020 Category: Pharmaceuticals Source Type: news

IDELVION(R) Becomes First and Only Factor IX Therapy with 21-Day Prophylactic Dosing
European Medicines Agency Approves 21-Day Dosing Intervals for Adult Haemophilia B Patients HATTERSHEIM, Germany, July 3, 2020 -- (Healthcare Sales & Marketing Network) -- Global biotherapeutics leader CSL Behring announced today that the European Medi... Biopharmaceuticals, Regulatory CSL Behring, IDELVION , albutrepenonacog alfa, Factor IX, haemophilia B (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - July 3, 2020 Category: Pharmaceuticals Source Type: news

Roche announces new data at the ISTH 2020 Congress, demonstrating ongoing commitment to advancing care for people with haemophilia A
Basel, 29 June 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data from its haemophilia A clinical programme will be presented at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Congress on 12-14 July 2020. Data will include updated safety results from the phase IIIb STASEY study of Hemlibra ® (emicizumab) and new results from the phase III HAVEN 5 study of Hemlibra. Data will also include insights into the impact of living with haemophilia A. Spark Therapeutics (a member of the Roche Group) will also present data from the initial dose cohorts of its phase I/II SPK-8011 gene ther...
Source: Roche Media News - June 29, 2020 Category: Pharmaceuticals Source Type: news

Roche announces new data at the ISTH 2020 Congress, demonstrating ongoing commitment to advancing care for people with haemophilia A
Basel, 29 June 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data from its haemophilia A clinical programme will be presented at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Congress on 12-14 July 2020. Data will include updated safety results from the phase IIIb STASEY study of Hemlibra ® (emicizumab) and new results from the phase III HAVEN 5 study of Hemlibra. Data will also include insights into the impact of living with haemophilia A. Spark Therapeutics (a member of the Roche Group) will also present data from the initial dose cohorts of its phase I/II SPK-8011 gene ther...
Source: Roche Investor Update - June 29, 2020 Category: Pharmaceuticals Source Type: news

Why four years is important for BioMarin, hemophilia patients
An experimental gene therapy for hemophilia A from BioMarin Pharmaceutical Inc. — promising a one-shot fix for the genetic cause of the bleeding disorder — continues to hold patients' bleeds to less than one a year after four years. The results of the ongoing study are important as San Rafael-based BioMarin's (NASDAQ: BMRN) gene therapy, called Roctavian and known scienti fically as valoctogene roxaparvovec, nears an Aug. 21 decision date from the Food and Drug Administration. If approved, it… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - June 1, 2020 Category: Biotechnology Authors: Ron Leuty Source Type: news

2 Bay Area companies ramp up in quests for one-shot-and-done gene therapies
As BioMarin Pharmaceutical Inc. eyes a groundbreaking approval this summer of a one-shot-and-done treatment for hemophilia A patients, it and another Bay Area gene therapy company made big steps Monday. BioMarin (NASDAQ: BMRN) of San Rafael paired with one-year-old Swiss company DiNAQOR AG to develop gene therapies to treat rare genetic heart diseases and Adverum Biotechnologies Inc. (NASDAQ: ADVM) of Redwood City reported that the latest cohort in its early-stage clinical trial of patients with… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - May 4, 2020 Category: Biotechnology Authors: Ron Leuty Source Type: news

Sports participation and sports injuries in Dutch boys with haemophilia - Versloot O, Timmer M, de Kleijn P, Schuuring M, van Koppenhagen CF, van der Net J, Fischer K.
INTRODUCTION: Sports participation in children with haemophilia is generally considered to be associated with increased injury risk, which is generally considered highest in severe haemophilia. AIM: To assess sports participation according to age a... (Source: SafetyLit)
Source: SafetyLit - April 7, 2020 Category: International Medicine & Public Health Tags: Age: Adolescents Source Type: news

FDA Approves Sevenfact [coagulation factor VIIa (recombinant)-jncw] for Treatment and Control of Bleeding Episodes Occurring in Adult and Adolescent Hemophilia A and B Patients with Inhibitors
LOUISVILLE, Ky., April 6, 2020 /PRNewswire/ -- HEMA Biologics, LLC, ( " HEMA Biologics " ) today announced that the U.S. Food and Drug Administration (FDA) approved Sevenfact® [coagulation factor VIIa (recombinant)-jncw] as the first new bypassing... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - April 6, 2020 Category: Drugs & Pharmacology Source Type: news

HEMA Biologics(TM) Announces FDA Approval of SEVENFACT(R) [coagulation factor VIIa (recombinant)-jncw] for Treatment and Control of Bleeding Episodes Occurring in Adult and Adolescent Hemophilia A and B Patients with Inhibitors
First new bypassing agent approved for the treatment and control of bleeding episodes in hemophilia A and B patients with inhibitors in over 2 decades Median number of infusions required to achieve bleeding control in the first 12 hours was 1 (225 mcg/k... Biopharmaceuticals, FDA HEMA Biologics, SEVENFACT, coagulation factor VIIa, hemophilia (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - April 6, 2020 Category: Pharmaceuticals Source Type: news

FDA Approves Sevenfact (coagulation factor VIIa [recombinant]-jncw) for Adults and Adolescents with Hemophilia A or B and Inhibitors
April 01, 2020 -- The U.S. Food and Drug Administration today approved Sevenfact [coagulation factor VIIa (recombinant)-jncw] for the treatment and control of bleeding episodes occurring in adults and adolescents 12 years of age and older with... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - April 1, 2020 Category: Drugs & Pharmacology Source Type: news

Health Tip: Coping With Winter Nosebleeds
-- Wintry climates and cold viruses can lead to frequent nosebleeds, says the National Hemophilia Foundation. To prevent nosebleeds during winter, the foundation suggests: Use a humidifier to moisturize the air. Use a nasal saline spray or... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - January 10, 2020 Category: General Medicine Source Type: news

Gene therapy shown to offer long-term benefits for people with Haemophilia A
(Queen Mary University of London) A breakthrough gene therapy treatment for Haemophilia A has been shown to offer long-term benefits that have already transformed the lives of 13 men in the UK. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 6, 2020 Category: International Medicine & Public Health Source Type: news

Gene Therapy May Be Long-Term Cure for Type of Hemophilia
Title: Gene Therapy May Be Long-Term Cure for Type of HemophiliaCategory: Health NewsCreated: 1/2/2020 12:00:00 AMLast Editorial Review: 1/3/2020 12:00:00 AM (Source: MedicineNet Chronic Pain General)
Source: MedicineNet Chronic Pain General - January 3, 2020 Category: Anesthesiology Source Type: news

Gene Therapy May Be Long-Term Cure for Type of Hemophilia
THURSDAY, Jan. 2, 2020 -- A new gene therapy appears to serve as a functional cure for the most common type of hemophilia, early clinical trial results indicate. Patients who received the one-time intravenous therapy continue to have a more than... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - January 2, 2020 Category: General Medicine Source Type: news

Hepatitis gene therapy leads Bay Area trio of year-end FDA approval requests
Approaching the end of long drug-development journeys, these three Bay Area companies formally asked the FDA to approve potential treatments for hemophilia A, anemia in chronic kidney disease patients and migraines. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - December 23, 2019 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

Hepatitis gene therapy leads Bay Area trio of year-end FDA approval requests
Approaching the end of long drug-development journeys, these three Bay Area companies formally asked the FDA to approve potential treatments for hemophilia A, anemia in chronic kidney disease patients and migraines. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - December 23, 2019 Category: Biotechnology Authors: Ron Leuty Source Type: news