Latest Clinical Trials
Generation Bio is working to “create the next generation of gene therapy” that will enable the redosable, durable expression of missing proteins.
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…
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…
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…
Researchers worked to better understand and manage patients with mild or moderate hemophilia, who can be an underrepresented and underserved population.
Hemophilia Clinical Insights
An increasing number of studies support the use of gene therapy in patients with hemophilia. However, several factors restrict its wide application.
We examine the relatively new prophylactic hemophilia A drug, efmoroctocog alfa, and how discuss the data on how well it works.
The clinical management of acquired hemophilia A still puzzles physicians and hematologists globally, and early diagnosis is difficult.
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