Latest Clinical Trials
Excessive bleeding in patients with undiagnosed acquired hemophilia A can be lethal.
Patients with nonsevere hemophilia should still be managed effectively to prevent a deterioration in their quality of life.
Patients should be placed front and center in any discussions about hemophilia therapeutic options.
Scientists suggest immunosuppression therapy may help reduce formation of inhibitors with factor VIII replacement therapy in hemophilia.
Although often overlooked, sexual dysfunction can occur in patients with hemophilia, negatively impacting quality of life.
Questionnaires assessing the quality of life of patients with hemophilia reveal that a large percentage of individuals struggle on a daily basis.
The mainstay of hemophilia treatment is impeded by a stubborn problem that can sometimes drastically reduce its efficacy.
Research shows thrombin generation assays can monitor the efficacy of hemophilia A treatment in a way that the mere monitoring of plasma FVIII activity cannot.
Rare disease advocates are marking the 40th anniversary of the landmark Orphan Drug Act, which has led to over 1100 approvals for new therapies.
The Bespoke Gene Therapy Consortium (BGTC) is an ambitious public-private partnership that aims to develop gene therapies for ultrarare diseases.
The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
Generation Bio is working to “create the next generation of gene therapy” that will enable the redosable, durable expression of missing proteins.
Rare Care Podcast
The Pursuit of Redosable Gene Therapies for Hemophilia with Dr. Doug Kerr