hemophilia A

Oct 23, 2024

Plasma protein therapeutics are revolutionizing the landscape of modern medicine by harnessing the healing potential of the human body. Derived from donated blood plasma, these life-saving treatments are packed with antibodies, clotting factors, and other essential proteins that help manage a wide array of conditio...

Jul 14, 2023

Hemophilia A is a rare genetic bleeding disorder that primarily affects males. Hemophilia A is the most common type of hemophilia, accounting for approximately 80% of all cases. It is estimated to occur in about 1 in 5,000 to 10,000 male births. As per the Centers for Disease Control (CDC) (2020), the estimated pre...

Jul 04, 2023

FDA Approves First Gene Therapy for Severe Hemophilia A BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that the US Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the tr...

Jun 09, 2023

The cell and gene therapy market has seen a revolutionary transition in recent years, with advancements in scientific research and novel methods of treatment driving a rise in development activities. This has resulted in an increase in the number of cell and gene therapy choices available to patients suffering from...

Nov 07, 2022

Hemophilia A treatment scenario before the launch of HEMLIBRA Despite the ups and downs throughout history, the management of hemophilia A patients substantially improved over the past 40 years. The groundbreaking discovery of cryoprecipitate in 1964 marked the beginning of the modern progression of hemophilia t...

Sep 27, 2022

Daiichi Sankyo Receives the First Approval for its Blood Cancer Drug Ezharmia Daiichi Sankyo has received the first global approval for Ezharmia, a first-in-class dual EZH1 and EZH2 inhibitor for the treatment of patients with relapsed or refractory adult T-cell leukemia/lymphoma (ATL). The Japanese Ministry of ...

Aug 19, 2022

There has been a renaissance in gene therapy attempts, spurred partly by the discovery and understanding of novel gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that may be designed to carry DNA to target cells and has sparked considerable interest in the area, particularly in clinical...

Jul 12, 2022

Novo Nordisk Reports Phase III Results of Concizumab Drug for Hemophilia A or B Novo Nordisk has announced Phase III results for their concizumab drug for hemophilia A or B, demonstrating efficacy in preventing bleeding events and paving the way for regulatory filings later this year. Concizumab, an anti-tissue ...

Jun 28, 2022

Ipsen to Buy Biopharmaceutical Company Epizyme for USD 247 Million The restructuring of French manufacturer Ipsen has progressed with a takeover agreement for US competitor Epizyme and its cancer drug Tazverik in a deal for slightly less than USD 250 million. Ipsen is proposing USD 1.45 per share for Epizyme, va...

Mar 21, 2022

The universality of mobile technology has proven to be extremely beneficial, specifically in recent years. The potential of improving medical care delivery, and the potential for instantaneous and two-way transfer of information has turned out to be a revolutionary step. The following article indicates the importan...