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FDA awards LAMA2-CMD therapy MDL-101 orphan drug status
MDL-101, Modalis Therapeutics’ epigenetic editing therapy for LAMA2-related congenital muscular dystrophy (LAMA2-CMD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The designation is intended to encourage the development of therapies for serious or life-threatening rare diseases, which are those affecting fewer than 200,000 people in the U.S. The designation…
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Duchenne affects my entire life, down to a DIY project
I’m a mom to seven children: Lexi, 23; Max, 18; Chance, 17; Rowen, 15; Charlie, 13; Mary, 10; and Callie, 2. As part of that job, I’m also a caregiver to Max, Rowen, and Charlie, who all have Duchenne muscular dystrophy (DMD). Additionally, I’m a chronic DIYer (do-it-yourselfer). Yes, you read that correctly. Perhaps you’re…
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Mouse model reveals liver involvement in muscular dystrophy
A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 — the most prevalent form of adult-onset muscular dystrophy — provides insight into why patients develop fatty liver disease and display hypersensitivity to medications, making treatment difficult. The new model opens avenues for screening new medications for liver toxicity prior to patient…
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A new beginning of shared purpose at Shalom Medcare
Since September, my life has moved in an exciting new direction, starting with becoming a marketing communications assistant at Shalom Medcare (SMC), a medical transport company here in Singapore. In case you’re wondering, yes, I realize it’s amusing that the company and I share the name “Shalom” — something that’s been a conversation starter ever…
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DM1 progression varies with sex, age at disease onset: Study
The progression of myotonic dystrophy type 1, known as DM1, differs according to sex and age at symptom onset, according to the findings of a four-year study, but the researchers nonetheless reported “notable variability” with patients’ general decline. The study, by researchers in the Netherlands, focused on changes over time in muscle strength, hand grip,…
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Dallas Community Comes Together to Raise $100,000 for CureDuchenne at Champions in Dallas Dallas’ Finest Unites to Advance Innovative Research and Care for Duchenne Muscular Dystrophy
Dallas, TX – October 22, 2024– The Dallas community came together in an extraordinary show of support at Champions in Dallas on October 3, 2024, raising $100,000 for CureDuchenne, a leading nonprofit dedicated to finding a cure for Duchenne muscular dystrophy. Held at the stunning DHV Artworks, the event was a night filled with culinary…
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Changing society and its views on disability, one column at a time
Like me, my colleagues here at Bionews, the parent company of this website, write about their lives with chronic illness. In our columns, “we make a promise to our rare disease communities to deliver trustworthy information to help educate, engage, and champion the patient voice.” These words appear on the Bionews website, and they’re the…
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EMA agency continues to oppose renewing Translarna’s approval
The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency — again maintained its recommendation not to renew conditional marketing authorization for Translarna (ataluren), a treatment for Duchenne muscular dystrophy (DMD) caused by nonsense mutations. The European Commission, which has final say over medication approvals in the European Union,…
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I wish all of my sons could have played football
I sat in the front row of the football stadium, watching my second-oldest son, Chance, 17, play football. It’s his senior year, and I’ve been front and center at each game. That front row has the accessible seating, which helps because Chance has three brothers living with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15,…
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CIFFREO Data Discussion Video
Watch the video HERE Pfizer recently provided this video updating the results from their Phase 3 micro-dystrophin gene therapy trial, CIFFREO, which were presented on October 12, 2024 at the World Muscle Society Conference in Prague. In this video, Francesco Muntoni, Chair of the CIFFREO trial steering committee, and Dan Levy, who leads DMD Clinical…
