hemophilia

Apr 07, 2025

Over the past three years, the FDA has approved six new hemophilia treatments, including three gene therapies. Now, Sanofi’s Qfitlia (fitusiran) enters the competitive hemophilia therapeutics market, distinguishing itself as the only therapy approved for all types of hemophilia. Unlike most existing treatments, Qfi...

Apr 01, 2025

Sanofi’s Qfitlia Becomes First US-Approved Therapy for Hemophilia A or B The FDA has approved Qfitlia (fitusiran), the first antithrombin-lowering therapy for routine prophylaxis to prevent or reduce bleeding episodes in hemophilia A or hemophilia B patients (ages 12+) with or without inhibitors. Approval is bas...

Nov 21, 2022

RNA interference (RNAi) is a fundamental gene-silencing pathway in eukaryotic cells that involves an enzyme called dicer cleaving long pieces of double-stranded RNA into shorter fragments called siRNAs that can cleave complementary mRNA sequences with the help of the RISC complex and argonaute. RNA interference ...

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...

Nov 08, 2021

Hemophilia is an inherited rare disorder where blood doesn’t clot in the regular way because the person affected doesn’t make enough blood-clotting proteins (clotting factors). Without these factors, patients cannot stop bleeding when they are injured. Patients with more severe forms of the disease can experience s...

Mar 17, 2021

Bleeding disorders, also known as coagulopathy, are a group of conditions in which the blood cannot clot properly due to the missing or defective protein. Bleeding Disorder can affect a person irrespective of his genders, ages and race and the person is likely to bleed more than the average person. A minor cut or s...

Mar 15, 2021

An X-linked recessive disorder, Hemophilia occurs predominantly in males with females being the carriers. Patients diagnosed with Hemophilia usually lack one of the two proteins essential for the blood coagulation cascade namely factor VIII (FVIII) and factor IX (FIX). On the basis of the different blood coagulatio...

Mar 08, 2021

A Royal Disease: Hemophilia  During the 19th-century British monarch's son Leopold, Duke of Albany slipped and died of blood loss. Not only once, but Queen Victoria’s grandsons died at a comparatively younger age. Thus, it was thought that male descendants of the royal family are cursed. Hence, the assumed ...