News

Although obese people with hemophilia — as well as their spouses and caregivers — are aware of the risks of excessive weight, fewer than half are actively taking steps to lose weight, a new study shows. Better support and education about weight management would be beneficial for those patients, the…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

Ixinity remains readily available to treat people with hemophilia B despite the current COVID-19 outbreak, Medexus Pharmaceuticals has announced. The company, which acquired Ixinity from Aptevo Therapeutics, also said it will continue working to ensure continued supply throughout the pandemic. “We would like to reassure the patients…

A Phase 3 clinical trial investigating gene therapy candidate AMT-061 (etranacogene dezaparvovec) has reached its target of dosing 50 participants with moderate or severe hemophilia B. In fact, according to AMT-061 developer uniQure, 54 patients have been given the one-time dose of AMT-061 in the HOPE-B…

Gene therapy delivering the blood clotting factor VIII (FVIII) — whose lack causes hemophilia A — into the joints did better at protecting against hemophilic arthropathy (joint damage) than did administration into the bloodstream, a study in mice suggests. Its findings support the potential use of FVIII injected directly into…

Endothelial cells — those that line the inside of blood vessels — derived from stem cells of people with hemophilia A and modified to produce functional factor VIII were able to ease blood loss in a mouse model of the disease, a study reported. These findings support the potential of induced…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) — launched…

Some $80.3 million in financing will support the first clinical trial of Sigilon Therapeutics’ new hemophilia A cell therapy candidate, SIG-001, the company said. The trial is expected to begin by June. Last August, the U.S. Food and Drug Administration (FDA) granted the potential cell therapy…

Novo Nordisk has paused three clinical trials evaluating concizumab, its experimental treatment candidate for hemophilia A and B, due to safety concerns. The three Explorer studies — the Phase 2 Explorer 5 (NCT03196297), and the global Phase 3 Explorer 7 (NCT04083781) and Explorer 8 (NCT04082429)…

Cells derived from hemophilia A patients — genetically reprogrammed to produce a functional clotting factor VIII (FVIII) that’s missing or not working in people with the blood disease — were successfully grafted into hemophiliac mice, restoring blood levels of FVIII and significantly improving…