SerpinPC, an experimental hemophilia therapy, at high dose safely reduced overall bleed rates by up to 88% and spontaneous joint bleeds by up to 94% in adults with severe hemophilia A and B, who were not using preventive treatment, according to top-line results from a Phase 2a…
Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…
A report describes — for the first time, according to study authors — the case of a girl who has both hemophilia A and C. To date, only 10 such cases had been documented in medical literature, all occurring in men. The case report authors highlight the importance of…
Ten U.K. families who are members of the Haemophilia Society have the chance to enjoy a free, virtual Camp in the Cloud experience offered by Over The Wall, a nonprofit specializing in activity camps for children living with serious disorders. The places, each worth £150 (about $206), are available on a first-come,…
The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…
Women who are carriers for hemophilia — meaning they can pass the disease-causing mutation to their children — think a more individually tailored, information-rich healthcare approach could improve the experience of getting or being pregnant, a small study found. “Healthcare professionals could use these insights to adapt their consultations to…
Preventive treatment with marzeptacog alfa activated (MarzAA) can reduce bleed frequency substantially in men with hemophilia A or B who developed inhibitors to conventional replacement therapies, according to data from a Phase 2 clinical trial. The findings were reported in the study, “Subcutaneous engineered factor…
Treatment with the investigational gene therapy Roctavian (valoctocogene roxaparvovec) can reduce bleeding frequency and the use of replacement therapies, while maintaining or improving the quality of life of people with severe hemophilia A for up to five years, updated Phase 1/2 trial data show. “These…
Bleeding disorder patients and their supporters are encouraged to participate on Sept. 18 in Unite Day, a national virtual event aimed at celebrating the community and underscoring the need for treatment for all. Unite Day complements participation in the annual Unite for Bleeding Disorders Walk, both presented by the…
The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…
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