Latest Clinical Trials
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.
What is acquired hemophilia A, and why is it underdiagnosed and undermanaged? We attempt to answer these questions and more.
We discuss the importance of pain management in hemophilia care and the effort of Italian doctors to arrive at a consensus on this issue.
We compare hemophilia treatment strategies and approaches among different countries, particularly Japan and China.
We explore how the treatment landscape of hemophilia is changing as scientists race to find a permanent cure.
An increasing number of studies support the use of gene therapy in patients with hemophilia. However, several factors restrict its wide application.
Generation Bio is working to “create the next generation of gene therapy” that will enable the redosable, durable expression of missing proteins.
When it comes to rare disorders, Israel—a New Jersey-sized nation of 9.3 million that’s made headlines this year for its highly successful coronavirus vaccination campaign—is clearly an emerging powerhouse.
Under COVID-19’s lingering shadow, the National Organization of Rare Disorders (NORD) has presented its 2021 Rare Impact Awards to 24 pharmaceutical firms, researchers, politicians, and nonprofit groups for their efforts over the past year on behalf of patients with such illnesses.
As the super-contagious Delta variant of COVID-19 sweeps across the globe, driving up infection rates as well as deaths, top US health officials are urging everyone 12 and older—including the country’s 30 million or so rare disease patients—to get vaccinated quickly.
Rare Care Podcast
The Pursuit of Redosable Gene Therapies for Hemophilia with Dr. Doug Kerr