In a Phase 1/2 dose-finding study called B-AMAZE, FLT180a gene therapy from Freeline Therapeutics brought about sustained levels of factor IX, the clotting factor that is missing in people with hemophilia B, and this translated into fewer bleeds each year. Levels of factor IX within the normal range…

As a woman living with hemophilia, I’ve always understood that hemophilia is a man’s world. But I’ve also discovered that the men inhabiting this world are kind, compassionate superheroes with massive struggles of their own. My first National Hemophilia Foundation conference was in 2018 in Orlando, Florida. More than 2,800…

The European Medicines Agency (EMA) has approved an accelerated assessment request for etranacogene dezaparvovec, an experimental gene therapy for hemophilia B. The decision means that, once an application is submitted seeking approval for marketing authorization of etranacogene dezaparvovec, it will be reviewed more quickly than normal — which…

Physical activities with a high risk of a collision are linked with a greater risk of bleeding in people with moderately severe hemophilia A, a six-month U.S. study reports. This risk was higher for people using on-demand versus preventive replacement factor VIII (FVIII) therapies, the researchers found, and they suggested…

Amarna Therapeutics has secured €5 million (about $5.6 million) in new funding, which it plans to use for developing its lead product, AMA005, an investigational gene therapy that aims to restore blood clotting in people with hemophilia B. The goal is to advance the company’s hemophilia B program…

A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical…

Canadian Blood Services (CBS) has awarded a three-year tender — an exclusive bulk purchase agreement aiming to minimize medication cost — to Takeda Canada for the purchase of Adynovate, an injectable replacement therapy for the prevention and treatment of bleeding episodes in people with hemophilia A. This…

Some years ago, when my youngest son, “MacDonald the Younger,” was about 7, he had an internal bleed into his right knee just before Thanksgiving. I was scheduled to lead a “Thanks Living” service that Tuesday, but we quickly realized we would be celebrating the holiday in a hospital…

Julian was only a baby when he received his hemophilia diagnosis, and it came as a big shock to me. I remember how much I enjoyed the newness of having a beautiful son who looked perfect. Then, the first bruise appeared. Julian has severe hemophilia A, also known as…

In an unexpected turn, Catalyst Biosciences is discontinuing the clinical development of marzeptacog alfa activated (MarzAA), its experimental under-the-skin therapy for hemophilia A and B patients with inhibitors, which was being evaluated in an international Phase 3 clinical trial. “Based on several factors including a recently updated feasibility assessment,…