Uncategorized

This article was provided by our partner, the Muscular Dystrophy Association. It has been reviewed by Bionews for accuracy and relevance. The views and opinions expressed are those of the author and do not necessarily reflect the views of Bionews or Muscular Dystrophy News Today. I grew up being educated on how I could help my…

Know More

My dear friend Colin Rensch had a saying: “If you think you can, then of course you can.” I met Colin because I live with limb-girdle muscular dystrophy, and he lived with Duchenne muscular dystrophy. Colin, who defined passion, purpose, and positivity, passed away last month at the age of 32. I’ve written in the…

Know More

It started long before I had the words to explain it. At 9 years old, I noticed small things: I struggled to lift my arms the same way as my friends, I grew tired more quickly, and others were much faster than me. But at that age, it was easy to brush those things off.…

Know More

Quest Media is an innovative, adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and members of the neuromuscular disease community about topics that matter to them and to the larger community of individuals with disabilities. With so many valuable…

Know More

I have been writing here about my family since August 2021. My husband, Jason, and I share seven children: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max, Rowen, and Charlie have Duchenne muscular dystrophy (DMD). In my first column, I shared that my husband and I were…

Know More

For many people living with generalized myasthenia gravis (gMG), the journey is not just defined by symptoms—it’s shaped by uncertainty. Uncertainty in diagnosis. Uncertainty in treatment. And for a significant subset of the community, uncertainty in whether therapies designed for others will work for them at all. That’s why moments like this matter. The recent…

Know More

Entrada Therapeutics, who received early funding from CureDuchenne to advance their Endosomal Escape Vehicle Platform, today announced positive initial data from their Phase 1/2 ELEVATE-44-201 study in DMD patients amenable to skipping exon 44. Read press release HERE The post Entrada Therapeutics announced positive initial data from their Phase 1/2 ELEVATE-44-201 study in DMD patients…

Know More

Epicrispr Biotechnologies is partnering with Forge Biologics to support the development of EPI-321, Epicrispr’s one-time epigenetic treatment candidate for facioscapulohumeral muscular dystrophy (FSHD). The treatment uses epigenetics — chemical modifications that can alter gene activity without changing a gene’s DNA sequence — to turn off the abnormally-activated gene that underlies FSHD. According to the developer,…

Know More

Motherhood is an incredible journey, layered with joy, growth, challenges, victories, and an abundance of love. For moms living with neuromuscular disease, the role of motherhood can sometimes entail additional layers, requiring extra patience, adaptability, and resilience. But, with a beautiful harmony, those very traits are often strengthened through daily life with a disability and…

Know More

Entrada Therapeutics has announced positive topline data from Cohort 1 of the double-blind, placebo-controlled, multiple ascending dose portion of the Phase 1/2 ELEVATE-44-201 study evaluating ENTR-601-44 in ambulatory participants ages 4–20 living with Duchenne muscular dystrophy amenable to exon 44 skipping. ENTR-601-44 is an investigational therapy combining their cell penetrating peptide with an exon skipping…

Know More