BioMarin Receives Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec Gene Therapy to Treat Adults with Severe Hemophilia A
European Commission Approval Expected Q3 2022 1st Gene Therapy for Treatment of Hemophilia A Recommended for Approval in Europe More than 20,000 Adults with Severe Hemophilia A in BioMarin Territories Across Europe, the Middle East and Africa Conf... Biopharmaceuticals, Regulatory BioMarin Pharmaceutical, Valoctocogene Roxaparvovec, Hemophilia (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - June 24, 2022 Category: Pharmaceuticals Source Type: news

Debated: Nonfactor Versus Gene Therapy for Hemophilia Debated: Nonfactor Versus Gene Therapy for Hemophilia
The question debated was, should patients with hemophilia A stick with effective nonfactor therapy or join a clinical trial for a potential cure with gene therapy?Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - June 23, 2022 Category: Consumer Health News Tags: Hematology-Oncology News Source Type: news

For haemophiliacs, most of the world is still living in the dark ages
A sufferer explains how the differences in treatment between the UK and his native India highlight a global medical apartheidLike the Hindu deity Krishna, I was born with blue skin. My body bruised at the trauma of simply being held. And so the family arranged for a ritual to appease the gods.Haemophilia is a genetic blood disorderthat makes it very hard for the body to stop bleeding. If your haemophilia is severe like mine, you bleed spontaneously, without an injury or known cause. A handshake once took me to A&E.To stop bleeding, you need clotting injections. In much of the developing world, these injections are avai...
Source: Guardian Unlimited Science - June 14, 2022 Category: Science Authors: Tarun Gidwani Tags: Health Global development Genetics Women NHS Science Society Pharmaceuticals industry Business Source Type: news

For people with haemophilia, most of the world is still in the dark ages
Differences between the UK and India in treating the blood-clotting disease highlight a global medical apartheidLike the Hindu deity Krishna, I was born with blue skin. My body bruised at the trauma of simply being held. And so the family arranged for a ritual to appease the gods.Haemophilia is a genetic blood disorderthat makes it very hard for the body to stop bleeding. If your haemophilia is severe like mine, you bleed spontaneously, without an injury or known cause. A handshake once took me to A&E.To stop bleeding, you need clotting injections. In much of the developing world, these injections are available only to...
Source: Guardian Unlimited Science - June 14, 2022 Category: Science Authors: Tarun Gidwani Tags: Health Global development Genetics Women NHS Science Society Pharmaceuticals industry Business Source Type: news

An infant with severe hemophilia A with intracranial hemorrhage mistaken for child abuse: a case report - Youn JW, Shim YJ, Byun JC, Kwon SM.
[The publisher has not provided an abstract for this article.] Language: en... (Source: SafetyLit)
Source: SafetyLit - June 13, 2022 Category: International Medicine & Public Health Tags: Age: Infants and Children Source Type: news

BioMarin delays refiling approval request for hemophilia gene therapy
The once-and-done gene therapy, initially rejected by the FDA in summer 2020, could potentially provide a genetic fix for a bleeding disorder that today can require multiple prophylactic injections a week. (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - May 31, 2022 Category: Health Management Authors: Ron Leuty Source Type: news

' It's an Amazing Time to Be a Hemophilia Provider'' It's an Amazing Time to Be a Hemophilia Provider '
New drugs such as emicizumab (Hemlibra) are transforming the lives of patients with hemophilia A, and more are on their way."It ' s an amazing time to be a hemophilia provider," says Alice D. Ma, MD.Medscape Medical News (Source: Medscape Hematology-Oncology Headlines)
Source: Medscape Hematology-Oncology Headlines - May 27, 2022 Category: Cancer & Oncology Tags: Hematology-Oncology News Source Type: news

Tanzania: Govt Unveils Hemophilia Clinic At BMH
[Daily News] THE government has unveiled a hemophilia clinic at the Benjamin Mkapa Hospital (BMH) in a bid to bring closer medication to people with the rare diseases in Central Zone and neighbouring regions. (Source: AllAfrica News: Health and Medicine)
Source: AllAfrica News: Health and Medicine - May 26, 2022 Category: African Health Source Type: news

Gene therapies can reduce treatment burden and costs for patients with blood disorders
Anew analysis found potential gene therapies for blood disorders like hemophilia, beta thalassemia, and sickle cell disease hold promise in dramatically reducing existing treatment burdens for patients, which could result in as much as $730,000 in savings to the health care system and increase a patient ’s income by as much as $9,500 in the year following treatment. (Source: The Catalyst)
Source: The Catalyst - May 18, 2022 Category: Pharmaceuticals Authors: Katie Koziara Tags: Medicines in Development Personalized Medicine Source Type: news

Roche pledges to extend commitment to the World Federation of Hemophilia Humanitarian Aid Program to 2028
Roche (SIX: RO, ROG; OTCQX: RHHBY) has extended its commitment to the World Federation of Hemophilia (WFH) Humanitarian Aid Program until the end of 2028. Roche’s prophylactic treatment will be provided to the WFH Humanitarian Aid Program to continue to treat as many as 1,000 people with haemophilia A in locations where there is little to no access to treatment. (Source: World Pharma News)
Source: World Pharma News - May 12, 2022 Category: Pharmaceuticals Tags: Featured Roche Business and Industry Source Type: news

Roche pledges to extend commitment to the World Federation of Hemophilia Humanitarian Aid Program to 2028
The renewed commitment will provide continued preventative (prophylactic) treatment to as many as 1,000 people withhaemophilia A in locations where there is little to no access tohaemophilia treatmentProphylactic treatment aims to prevent bleeds and allow people withhaemophilia to achieve quality of life comparable to non-haemophilic individuals. Access to this is particularly restricted in developing countries, with the limited healthcare resources reserved for emergency situations and acute bleedsMore than 940 people across 30 countries have already benefited from Roche ’s donations since the start of the partnership i...
Source: Roche Media News - May 10, 2022 Category: Pharmaceuticals Source Type: news

Gambia: Gambia Observes World Haemophilia Day
[The Point] In a bid to create more awareness through community outreach programmes on Haemophilia, Safe Motherhood and Haemophilia Foundation recently organised a day's sensitisation forum in observance of World Haemophilia Day. (Source: AllAfrica News: Health and Medicine)
Source: AllAfrica News: Health and Medicine - April 26, 2022 Category: African Health Source Type: news

Tanzania: Haemophilia Society Calls Govt to Set Budget for Treatment
[Daily News] THE Haemophilia Society of Tanzania (HST) is calling upon the government to begin setting a budget for the purchase of treatment products for patients with haemophilia and other blood diseases. (Source: AllAfrica News: Health and Medicine)
Source: AllAfrica News: Health and Medicine - April 25, 2022 Category: African Health Source Type: news

[Ad hoc announcement pursuant to Art. 53 LR] Roche reports good sales growth in the first quarter of 2022
Basel, 25 April 2022Group sales increase 11%1 at constant exchange rates (CER) and 10% in Swiss francsPharmaceuticals Divisionsales up 6%; continued strong sales of new medicines for severe diseases. Impact of biosimilars decreases as expected.Diagnostics Divisionsales grow 24% due to good momentum in base business and continued high demand for COVID-19 tests. After a strong first quarter, significant decline in COVID-19-related testing expected.Highlights in the first quarter:FDA approval of Vabysmo (severe eye diseases); CHMP recommends EU approval of Polivy combination (aggressive form of blood cancer), Tecentriq (early...
Source: Roche Media News - April 25, 2022 Category: Pharmaceuticals Source Type: news

[Ad hoc announcement pursuant to Art. 53 LR] Roche reports good sales growth in the first quarter of 2022
Group sales increase 11%1 at constant exchange rates (CER) and 10% in Swiss francsPharmaceuticals Divisionsales up 6%; continued strong sales of new medicines for severe diseases. Impact of biosimilars decreases as expected.Diagnostics Divisionsales grow 24% due to good momentum in base business and continued high demand for COVID-19 tests. After a strong first quarter, significant decline in COVID-19-related testing expected.Highlights in the first quarter:FDA approval of Vabysmo (severe eye diseases); CHMP recommends EU approval of Polivy combination (aggressive form of blood cancer), Tecentriq (early-stage non-small cel...
Source: Roche Investor Update - April 25, 2022 Category: Pharmaceuticals Source Type: news