After handing over the development of the gene therapy candidate SB-525 to Pfizer, Sangamo Therapeutics has now concluded its collaboration agreement with Biogen to advance and market gene therapies for Alzheimer’s and Parkinson’s diseases, and other neuromuscular and other neurological disorders. Among other goals, the…

Fear. Millions of people are living with insane amounts of fear due to the outbreak of the COVID-19 virus. This includes fear that the food supply will be limited, fear of catching the virus, and fear of lost income. There is a lot for our world to fear. But…

Gene therapy delivering the blood clotting factor VIII (FVIII) — whose lack causes hemophilia A — into the joints did better at protecting against hemophilic arthropathy (joint damage) than did administration into the bloodstream, a study in mice suggests. Its findings support the potential use of FVIII injected directly into…

Novo Nordisk has paused three clinical trials evaluating concizumab, its experimental treatment candidate for hemophilia A and B, due to safety concerns. The three Explorer studies — the Phase 2 Explorer 5 (NCT03196297), and the global Phase 3 Explorer 7 (NCT04083781) and Explorer 8 (NCT04082429)…

Like other countries, the Philippines is in a state of panic due to the COVID-19 outbreak. Schools have canceled classes well into mid-April. The possibility of lockdowns in various cities and municipalities is quickly becoming a reality. In the metropolitan Manila area where I live, authorities are about to prohibit…

I do not have hemophilia. My sons are the ones who live with a chronic bleeding disorder, and at first, I struggled to find something we might have in common. My wife and I are musicians, and the running question during my wife’s pregnancies was, “What if they are…

My 14-year-old son Caeleb is having trouble walking. Some days are better than others, but the bad days are filled with crazy pain. His right knee and ankle have sustained tremendous damage over the years from the joint bleeds that are a complication of hemophilia. His chronic…

Struggles with “mood and emotions” and feelings of social isolation or inadequate social support are more pronounced in young children with hemophilia and their parents than in age-matched healthy peers,  a study from Japan reports. But such “anxieties,” which affect quality of life, were less a concern in survey…

Growing up is exciting. Most young adults spend their late teens and early 20s meeting new people and making new friends. As we grow older, we get jobs, move away from home, and date. Some choose to get married and have kids. Inevitably, we also outgrow some old friendships.

It seems as if there is a day for everything: there’s National Wear Red Day, Boss’s Day, Rare Disease Day, Love Your Pet Day, and even National Margarita Day. And months are also observed for causes such as Blood Donor Month, Breast Cancer Awareness Month, and National Yoga Month. From…