SPK-8011 may be a safe treatment strategy for patients with severe or moderately severe hemophilia A, achieving an overall 97% reduction in annual bleeds, according to preliminary data from an ongoing Phase 1/2 trial. The trial (NCT03003533) is intended to explore the safety and efficacy of a single intravenous…
Hemlibra (emicizumab), the sole approved prophylactic treatment for hemophilia A, is now available to patients in developing countries via the World Federation of Hemophilia (WFH) Humanitarian Aid Program after Roche and its subsidiaries Chugai and Genentech joined the program. The donation will provide access to Hemlibra for…
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…
Clinical data from the ongoing Phase 2/3 trial data evaluating MarzAA (marzeptacog alfa activated) in patients with Hemophilia A or B with inhibitors continues to demonstrate the treatment’s potential to prevent bleedings. The most recent trial results were discussed at the 12th Annual Congress of the European Association for Haemophilia…
A single administration of AMT-061, an investigational gene therapy for people with severe and moderately severe hemophilia B, increased therapeutic levels of factor IX (FIX) in all patients enrolled in uniQure’s ongoing Phase 2b trial, the company announced. Ultimately, this trial’s purpose is to confirm the dose of AMT-061…
Maintaining high concentrations of Adynovate in the blood of hemophilia A patients decreases the number of bleedings and enables more patients to stop having bleeds, according to the first results of a Phase 3b/4 study by Takeda.
A Phase 3 clinical trial of treatment candidate AMT-061 for severe and moderately severe hemophilia B has treated its first patient. The open-label HOPE-B study (NCT03569891) is testing the safety and effectiveness of AMT-061 in adult men with hemophilia B. Patient recruitment is continuing for an estimated total of 56…
People with hemophilia may benefit from physiotherapy and rehabilitation programs intended to manage bleeding and joint disease (arthropathy), but more research is needed to better understand those programs — and their duration — that might be of most help, a review study says. The review, “Recent advances in musculoskeletal…
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history — how disorders such as spinal muscle atrophy (SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials,…
Despite having lower oral health scores, children with hemophilia showed no differences in oral health-related quality of life compared to children without the condition, a study found. The study, “Parent’s report on oral health‐related quality of life of children with haemophilia,” was published in Haemophilia. Hemophilia is a…
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