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CureDuchenne partners on World of Warcraft research fundraiser
CureDuchenne is partnering with Blizzard Entertainment, maker of the massively multiplayer online role-playing game World of Warcraft, on a fundraiser to help advance research into new treatments for Duchenne muscular dystrophy (DMD). Through Jan. 7, players of the video game will have the opportunity to adopt an in-game pet fox named Reven, who will accompany…
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My trip to the Idlewild amusement park was wild, but worth it
Idlewild, which is near Ligonier, Pennsylvania, is an old-style amusement park that’s been around since the days when the gentry from Pittsburgh took the train to their summer homes in the foothills of the Laurel Highlands. It’s a wonderful park for all ages, but it’s absolutely perfect for younger children like my grandkids, Iva Jane,…
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FDA Accepts Translarna NDA Resubmission for Review
A Letter From PPMD’s Pat Furlong I am thrilled to share that the FDA has officially accepted PTC Therapeutics’ New Drug Application (NDA) resubmission for Translarna (ataluren). This acceptance is an important step, meaning the FDA will now carefully evaluate the data to determine ataluren’s potential for approval. According to PTC, the NDA resubmission is…
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Flu Season Ready: Protecting Yourself and Your Family
With the start of school and the onset of winter, we can expect a rise in respiratory viruses. This year, it’s crucial to safeguard yourself, your children, and your family from all respiratory infections, particularly the more severe ones like influenza (flu), RSV, and COVID. Because respiratory muscles can be weakened in individuals with Duchenne…
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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…
