UniQure has taken major steps this year toward U.S. and European regulatory approval of AMT-060, its gene-therapy treatment for hemophilia B. In January, the U.S. Food and Drug Administration (FDA) gave AMT-060 breakthrough therapy designation for patients with severe hemophilia B. In April the European Medicines Agency (EMA) gave it PRIME designation. The designations…

In this video from the Hemophilia Federation of America, Kyle McCullough and Jesse Mayhan share what it’s like living with severe hemophilia. The boys discuss their treatment and what they tell friends when they ask about their condition. They also talk about what they’d like to…

Hemophilia is a disease where a person blood lacks a factor which enables it to clot. This can lead to severe bleeding which can be life-threatening if left untreated. According to the World Federation of Hemophilia, there are three types of hemophilia: inherited, sporadic, and acquired. MORE: Five…

Two gene therapies from Sangamo Therapeutics for hemophilia A and B have been granted special regulatory designations by the U.S. Food and Drug Administration (FDA). Sangamo said in a press release that the FDA has granted orphan drug status to SB-525  gene therapy for hemophilia A, providing the company with…

An experimental form of genome editing called clustered regularly interspaced short palindromic repeats or CRISPR/Cas9 could offer a faster and cheaper method of treating people with hemophilia, compared to the current DNA editing method. Because hemophilia is due to DNA mutations, the genome editing could be used to efficiently slice at the DNA,…

In recognition of the World Hemophilia Day, BDI Pharma employees company-wide united to raise awareness and money to support those affected by bleeding disorders. Events were held April 17 at the company’s South Carolina, Texas and Kansas sites, with employees joining remotely to raise money to support the WFH (World Federation of Hemophilia) by wearing…

In this simple animated video from CSL Behring US, hemophilia is explained in easy-to-understand language aimed at children and caregivers of newly diagnosed hemophilia patients. MORE: Explaining hemophilia A and hemophilia B The narrator shares the basic difference between hemophilia A and hemophilia B, and that…

Hemophilia is a rare disorder where a person’s blood lacks a factor which enables it to clot, leading to excessive bleeding that can become life-threatening. This bleeding can occur internally or externally. According to the World Federation of Hemophilia and the National Heart, Lung…