Tiny particles containing RNA led to successful delivery and production of factor VIII (FVIII) — the clotting protein that is missing or defective in people with hemophilia A — in a mouse model of the disease, according to a new study. The study, “Functionalized lipid-like…
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Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…
Prophylactic (preventive) treatment with Hemlibra (emicizumab) safely and effectively lowers bleeding rates in children and adolescents with severe hemophilia A, according to a real-life study from Israel. The therapy’s effectiveness was independent of the presence of inhibitors (antibodies) against synthetic formulations of factor VIII (FVIII) — the missing…
The U.S. Food and Drug Administration (FDA)’s request for two years of additional clinical trial data on Roctavian, a potential gene therapy for hemophilia A, took its developer — BioMarin — and others in the hemophilia community by surprise. That decision, announced in a complete response letter of…
The U.S. Food and Drug Administration (FDA) has pushed back by more than one year a decision on Roctavian, the potential first gene therapy for adults with hemophilia A. In a complete response letter that took the therapy’s developer, BioMarin by surprise, the FDA said it needed…
The National Hemophilia Foundation (NHF) has opened a grant program to support the local groups and chapters that work with bleeding disorder families, and have watched as their revenue sources dry up during the COVID-19 pandemic. Called the COVID-19 Fund Bridge Grant program, the effort is open to…
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…
Refixia or Rebinyn (nonacog beta pegol, or N9-GP), an approved replacement therapy for hemophilia B, is effective and has a good safety profile in previously untreated boys younger than 6 with severe to moderately severe disease, an early analysis of a Phase 3 trial indicates. In…
Novo Nordisk has resumed its Phase 3 clinical trials investigating concizumab as a potential treatment for hemophilia A and B, the company announced in a press release. The trials were paused in March due to the occurrence of non-fatal thrombotic (blood-clotting) events in three patients. In collaboration…
When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience. Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…
