Latest News
Survey: Patients With Hemophilia A Prefer Emicizumab to FVIII Prophylaxis
Novel Therapy Prevents Inhibitor in Hemophilia A Patients Receiving Recombinant FVIII Treatment
Case Report: An Unusual Clinical Presentation of Acquired Hemophilia A
Many Hemophilia Carriers Have Abnormal Bleeding Scores
Nanoparticle-Based Approach Shows Potential in Hemophilia
Clinicians Comfortable With Extended Half-Life Factor Products for Hemophilia
Long-Term Exposure to Prophylactic Drug for Hemophilia A Improves Clinical Outcomes
Hemophilia Does Not Increase Risk of HCC After Hepatitis C Virus Eradication
Consensus Statements Released for Pain Management in Hemophilia Patients
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
Guidelines for the Immunosuppressive Treatment of Acquired Hemophilia A
Researchers issued updated recommendations for the treatment of acquired hemophilia A based on new evidence.
Diagnosing and Managing Patients With Low Von Willebrand Factor
Researchers conducted a review of what we know about low von Willebrand Factor and how best to treat this disease.
Using Machine Learning to Predict Hemophilia A Severity
Machine learning is more advanced than most people think and has every potential to change the world, including the world of medicine.
The ‘Remarkable’ Breakthroughs That Have Transformed Hemophilia Treatment
Our knowledge of hemophilia is slowly moving forward, and new therapies are set to soon replace or complement existing ones. Indeed, if we would take a look back at medical history, we should be proud that any treatment for hemophilia exists at all; it used to be an incurable disease in which prevention of bruising…
Rare Care Podcast
The Pursuit of Redosable Gene Therapies for Hemophilia with Dr. Doug Kerr
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