The prevalence rate of Duchenne muscular dystrophy (DMD) in the United States has increased, largely due to better treatments that are helping patients live longer, while the number of new cases holds steady, a study reports. Touted as the first of its kind involving DMD, the Deerfield…
Last week’s column, “Uniting to Effect Positive Change,” focused on the bigger picture of advocacy. This form of advocating is about a larger movement and changing the society we live in. I have heard from many people wanting to get more involved. I cherish this because the time is…
Lithium chloride — once used as table salt — improved muscle size and strength in mouse models of limb-girdle muscular dystrophy D1 (LGMD1) by blocking a protein that is overactive in some people with this disease. Inhibiting this protein may be a way of correcting muscle weakness in these…
Biotechnology company Vertex has acquired Exonics Therapeutics, which focuses on using gene-editing technologies to treat Duchenne muscular dystrophy (DMD) — a move that is being hailed as a positive step toward getting these therapies into clinics. Exonics was co-founded in 2017 by CureDuchenne, a nonprofit organization…
Combining medical appointments with school, work, and finances can sometimes overwhelm me. My mom used to be the keeper of my schedule, but now it’s all up to me, and the pressure is on. The best way for me to reduce stress when everything seems to happen at…
Researchers at the University of California in Los Angeles (UCLA) have been awarded a $175,000 grant from Parent Project Muscular Dystrophy (PPMD) to make CRISPR/Cas9 as safe and effective as possible as a gene therapy tool to treat Duchenne muscular dystrophy (DMD). According to the researchers, their planned CRISPR/Cas9 gene…
I promised you that Sammy would be back. He came to this column on April 18 (see: “True Accessibility for All: No Roadblocks, Please“), and because he is my alter ego, I figured we’d be talking again at some point. The last time Sammy caught up with me, I…
Santhera Pharmaceuticals has entered into a collaboration agreement with researchers from the Biozentrum of the University of Basel in Switzerland to develop a new gene therapy for treating people with LAMA2-deficient congenital muscular dystrophy (LAMA2 MD). LAMA2 MD, also known as merosin-deficient congenital…
Having Duchenne muscular dystrophy is hard because of its progressive nature. I can no longer participate in activities the way I have in the past. As the disease advances, it is important for me to select friends carefully. Not everyone sees past the wheelchair and my weakness…
The Institute for Clinical and Economic Review (ICER) issued a draft report raising serious concerns about the cost-effectiveness of Sarepta‘s exon-skipping therapies Exondys 51 (eteplirsen), and the investigational golodirsen, as well as the corticosteroid Emflaza (deflazacort; by PTC Therapeutics), for treating…
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