Latest News
Patients With Hemophilia Often Given Potentially Inappropriate Medications
Despite Treatment, Hemarthroses Common in Patients With Severe Hemophilia
Study Shows Risk-Benefit of Emicizumab Unchanged in Hemophilia A
New Genotyping Strategy Could Identify Color Blindness in Hemophilia
Novel Technology Induces FVIII Production in Hemophilia A Rodent Model
Behavioral Change Techniques Support Patients With Hemophilia
Real-World Data Show Shortcomings of Prophylactic FVIII for Hemophilia
Emicizumab and Fc Fusion Protein Remain Effective for Hemophilia A
Efanesoctocog Alfa Shows VWF-Independent Results in Hemophilia A
Latest Clinical Trials
Features
Hemophilia May Benefit From Redosable Gene Therapy
Generation Bio is working to “create the next generation of gene therapy” that will enable the redosable, durable expression of missing proteins.
Israel Emerges as Global Powerhouse in Rare Disease Research
TEL AVIV, Israel — On the 6th floor of Schneider Children’s Hospital in the Tel Aviv suburb of Petah Tikva, 30 children with Alagille syndrome and 2 with lysosomal acid lipase deficiency (LAL-D) receive specialized treatment from a trio of pediatric hepatologists. Across town, at Sheba Medical Center’s Edmond and Lily Safra Children’s Hospital, pediatric…
NORD Recognizes Progress in Fight Against Rare Disease With 2021 Rare Impact Awards
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. The virtual ceremony was hosted by John Whyte, MD, MPH, chief medical officer at WebMD, with commentary…
FDA, CDC Urge COVID-19 Vaccines for Rare Disease Patients on NORD Webinar
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. Some 185 million people have been infected, and nearly 4…
Treatment Challenges in Mild to Moderate Hemophilia
Researchers worked to better understand and manage patients with mild or moderate hemophilia, who can be an underrepresented and underserved population.
Hemophilia Clinical Insights
Barriers Remain in Gene Therapy for Hemophilia Despite Clinical Advancements
An increasing number of studies support the use of gene therapy in patients with hemophilia. However, several factors restrict its wide application.
What We Know About Efmoroctocog Alfa in Hemophilia A
We examine the relatively new prophylactic hemophilia A drug, efmoroctocog alfa, and how discuss the data on how well it works.
Managing Acquired Hemophilia A: Challenges and Perspectives
The clinical management of acquired hemophilia A still puzzles physicians and hematologists globally, and early diagnosis is difficult.
Exploring Mortality Rates in Patients With Hemophilia A
We explore where we are today with regards to the mortality rates of patients with hemophilia A, as well as the impact of emicizumab.
Rare Care Podcast
The Pursuit of Redosable Gene Therapies for Hemophilia with Dr. Doug Kerr
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