My faith in God and my spiritual and practical gifts have helped me to find a purpose worth living for in a body and world that make it difficult. Because of Duchenne muscular dystrophy, I can solve all of the small inconveniences in front of me without understanding the…
While federal funding for fiscal year 2020 has not been finalized, 79 members of Congress have vowed to back Duchenne muscular dystrophy (DMD) research, patient support efforts and drug development. On its website, Parent Project Muscular Dystrophy (PPMD) heralded results of its annual bipartisan campaign, expressing optimism…
Whenever the topic of accessibility comes up, you can bet there’ll be great passion. We all want the right to access and partake in everything beautiful in life, yet so much of the world is unreachable to those of us with disabilities. In this column, I introduce my alter…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to pamrevlumab (FG-3019), FibroGen‘s potential anti-fibrosis treatment for Duchenne muscular dystrophy (DMD) patients. Pamrevlumab is a human monoclonal antibody that blocks the activity of the connective tissue growth factor (CTGF), a protein involved in tissue scarring (fibrosis) linked…
Living with Duchenne makes a lot of things difficult, including socializing with other people. Throughout my life, especially as the disease has progressed, it’s gotten harder to relate to people without physical disabilities. Even with the added difficulty, I’ve succeeded socially by carefully choosing the people I surround myself…
Audentes Therapeutics is expanding its pipeline of potential molecular therapies, expecting to address 80% of patients with Duchenne muscular dystrophy (DMD) and all with myotonic dystrophy type 1 (DM1). The treatment strategy, called vectorized exon skipping, uses a modified adeno-associated virus (AAV) to deliver…
A single dose of gene therapy based on CRISPR-Cas9 may successfully treat Duchenne muscular dystrophy (DMD) in the long term or even permanently, a preclinical study in mice shows. The findings shed light on safety factors that may affect the long-term efficacy of this gene therapy, and provide new evidence…
Come on! Is it essential to set goals in life? Do we need them? How can those of us who deal with a muscle-wasting illness push ourselves appropriately and live life to the fullest extent possible? I can tell you that it’s easy to fall into inaction if we…
Using a genetic engineering tool to restore dystrophin while raising levels of utrophin, a similar protein, leads to better improvements in muscle function than either approach alone, research in a mouse model of Duchenne muscular dystrophy (DMD) reports. The study, “The potential of utrophin and dystrophin…
In fifth grade, it was hard for me to grasp why some people could play handball, run, and join the flag football team, but I couldn’t. I gave more weight than I should have to the things that I could not do. Looking back, I’m not surprised I thought that…
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