Genentech recently announced the results from a second interim analysis of the STASEY study, designed to examine Hemlibra® as it relates to safety, efficacy and health-related quality of life in people with…
Spark Therapeutics recently released updated clinical study data on SPK-8011, the company’s investigational gene therapy candidate for hemophilia A.
SPK-8011 is administered via a one-time intravenous infusion, which is designed to elicit the…
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NHF has learned of the recent death of a patient who had been participating in a gene therapy clinical trial for a rare muscle disorder known as X-Linked Myotubular Myopathy (XLMTM), which occurs almost exclusively in males and is characterized…
CSL Behring recently announced that it has agreed to acquire exclusive global license rights from uniQure, to commercialize etranacogene dezaparvovec (AMT-061), an investigational gene therapy candidate for patients with severe and moderately severe…
Sanofi and Sobi, along with the World Federation of Hemophilia (WFH) and WFH USA recently announced as extension of the two companies’ support of the WFH Humanitarian Aid Program. The enhanced support includes an additional donation of up to 500…
The Partners in Bleeding Disorders Education Program has announced the launch of a new online learning module, “Accounting for Home Care: The Role of Treatment Logs in Bleeding Disorder Self-Management.”
Healthcare providers who complete the…
Objective:
To analyze real world rFVIIIFc patient characteristics and treatment interval patterns in patients with hemophilia A based on specialty pharmacy dispensing records.
Methods:
A retrospective…
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The Partners in Bleeding Disorders Education Program has announced the launch of two new online learning modules that speak to the history of hemophilia, with particular focus on early understanding of the disease and the evolution of treatment and…