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…
As I’ve started relying more on people for things I used to do on my own, it’s become easier to ask for help without feeling like a burden. This toxic thought has been a challenging mental hurdle, but it’s one we all deserve to get over. With compassion,…
Getting your son into a Duchenne muscular dystrophy (DMD) clinical trial can be difficult, given the narrow criteria for eligibility — like walking ability — associated with such trials. Even for families who qualify, the experience can be draining, said Pat Furlong, founder and CEO of Parent Project Muscular…
The journal Orphanet Journal of Rare Diseases has published a review highlighting the prevalence of Duchenne muscular dystrophy (DMD) as well as the disease’s costs and treatment patterns in North America and Europe. The analysis, “The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence…
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