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…
Preventive treatment with Kovaltry (octocog alfa) is safe and effective at preventing bleeds, including joint bleeds, for up to two years in people with hemophilia A, a long-term extension study shows. Findings were reported in the study “BAY 81-8973 demonstrated efficacy, safety and joint status improvement…
Struggles with “mood and emotions” and feelings of social isolation or inadequate social support are more pronounced in young children with hemophilia and their parents than in age-matched healthy peers, a study from Japan reports. But such “anxieties,” which affect quality of life, were less a concern in survey…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…
ClotChip, a portable device designed to quickly assess the ability of a person’s blood to clot, has been designated a breakthrough device by the U. S. Food and Drug Administration (FDA), its maker XaTek announced. This designation offers the company additional FDA guidance and the possibility of priority…
In an agreement expected to exceed $100 million, Medexus Pharmaceuticals has bought global commercial rights to Ixinity, an approved recombinant factor IX therapy for hemophilia B previously marketed by Aptevo Therapeutics. Sale terms include an upfront $30 million payment, plus up to $11 million in prospective…
In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…
In support of Bleeding Disorders Awareness Month in March, the Plasma Protein Therapeutics Association (PPTA) is providing a global outlet for patients who wish to share their experiences, advocate for therapy access, and thank plasma donors. The campaign — called “How Is Your Day?” — seeks to heighten awareness of…
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…
When given at low doses, extended half-life factor IX concentrates Alprolix (eftrenonacog alfa) and Idelvion (albutrepenonacog alfa) can prevent spontaneous bleeds in patients with severe hemophilia B, a study has found. The study, “A single centre retrospective study of low dose prophylaxis with…
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