Still Life with Duchenne Muscular Dystrophy

In the February 20th edition of the New York Times, Reed Abelson reports a “New Tack on a Muscle Disease.” The “new tack” is, in fact, a scaling back of ambition in muscular dystrophy (MD) research. Since identification of various genetic mutations that cause muscular dystrophies, gene transfer has been a logical goal. But because the gene is so large, and delivery to all of a patient’s muscles so difficult to achieve, gene transfer against MD has presented a formidable challenge to researchers.

What is so striking about Abelson’s article, in my view, is his suggestion that the ambition of a one-off cure has clouded the clinical and research community from recognizing and advancing interventions that extend life and improve its quality for MD patients. Abelson writes “the lack of interest in the mundane has… slowed progress in knowing what available therapies are the most useful.”

One researcher stated “for too long, I was stuck on [Duchenne] as the home run or nothing.” Though speculative, “home run” research deserves sanctuary in biomedical research, I regard a critical role for disease advocates, caregivers, and bioethicists to advocate on behalf of the quotidian needs of present day patients (and, I should add, gene transfer will likely play an contributions serving these quotidian needs). (photo credit: Evamol, “Still Life,” 2007)

2008 February 26 | Leave a comment

BibTeX

@Manual{stream2008-178,
    title = {Still Life with Duchenne Muscular Dystrophy},
    journal = {STREAM research},
    author = {Jonathan Kimmelman},
    address = {Montreal, Canada},
    date = 2008,
    month = feb,
    day = 26,
    url = {https://www.translationalethics.com/2008/02/26/still-life-with-duchenne-muscular-dystrophy-2/}
}

MLA

Jonathan Kimmelman. "Still Life with Duchenne Muscular Dystrophy" Web blog post. STREAM research. 26 Feb 2008. Web. 04 May 2024. <https://www.translationalethics.com/2008/02/26/still-life-with-duchenne-muscular-dystrophy-2/>

APA

Jonathan Kimmelman. (2008, Feb 26). Still Life with Duchenne Muscular Dystrophy [Web log post]. Retrieved from https://www.translationalethics.com/2008/02/26/still-life-with-duchenne-muscular-dystrophy-2/

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