In this video, a mother of two sons with Duchenne Muscular Dystrophy, both of whom now receive drug treatments, explains how she did not really worry about the disease progression; how there were no guarantees in expected timelines pre-treatment and there are none post.  She also talks about her son’s peers dying and the guilt associated with participating in a trial.

In this video, a mother of two sons with Duchenne Muscular Dystrophy and one with a primary immune deficiency talks her sons Austin and Max understanding their own life-limiting diagnosis, normalizing it within their family and the impact it has had on both boys.

In this video, a mother of two sons with Duchenne Muscular Dystrophy offers guidance on deciding to participate in a clinical trial. She asks, “what are you looking for- curative treatments vrs mitigating symptoms?”;  “What can your children stand – can they tolerate the tests required?”; “Can your family/marriage survive the lifestyle changes you may need to make to participate?”; “What if it is a trial with a placebo arm – can you handle your child getting the placebo?” She encourages asking lots of questions of the trial sponsor, doctors, and the disease community.

In this video, a mother of two sons with Duchenne Muscular Dystrophy talks about only one of her sons meeting the inclusion criteria and qualifying for the study as well as her naivety about how often timelines in studies change.  She explains a double-blind placebo study and how in her son’s study, all the participants began receiving the drug once evidence proved safety and efficacy. She talks about the FDA process and the importance of knowing the sponsors plan for the period of time between study completion and approval of the treatment.

In this video, a mother of two sons with Duchenne Muscular Dystrophy talks about finding out about a trial on Facebook, screening, and the whys of trial design.  She speaks about why it is important to enroll the right candidates in trial. She also discusses the misconception that you can “train” your child to meet the inclusion criteria, in this case a walk test.

In this video, a mother of two sons with Duchenne Muscular Dystrophy talks about the excitement and pressure families feel when there is the possibility of participating in a clinical trial and how that excitement can blind you to the important things like attending to the informed consent.

In this video, a mother of two sons with Duchenne Muscular Dystrophy talks how she felt is was advantage to be young and have no preconceived notions about her son’s diagnosis.

In this video, a mother of two sons with Duchenne Muscular Dystrophy talks about her oldest son, Austin’s diagnosis and how instinctually she knew he had an issue. She describes the unlikely occurrence of a spontaneous mutation and her son Max’s subsequent diagnosis via the thoughtful evaluation of a P.T.

In this video, a mother of two sons, Austin and Max with Duchenne Muscular Dystrophy and a son, James with a Primary Immune Deficiency, talks about evaluating her risk tolerance in finding a curative treatment for James. She talks about the barriers and biases when your child’s disease is considered a chronic, manageable condition and the limitations on the quality of her son’s life.