Nav: Home

Duchenne muscular dystrophy prevalence increases, while incidence remains steady

May 20, 2019

New York, NY, May 20, 2019--In the first study of its kind involving Duchenne Muscular Dystrophy (DMD) in the U.S., researchers from the Deerfield Institute found that while the number of new cases has remained stable, there has been an uptick in prevalence--largely attributed to enhanced treatments and longevity. The study, which is titled "Duchenne Muscular Dystrophy Prevalence in the U.S.: A Novel Incidence-Based Modeling Approach Using System Dynamics", is scheduled for Poster Session ll on Monday, May 20 at the ISPOR 2019 annual meeting in New Orleans.

DMD, a genetic disorder characterized by progressive muscle degeneration and weakness, is caused by an absence of Dystrophin, a protein that helps keep muscle cells intact. Symptom onset is in early childhood, typically between ages 3 and 5. The disease primarily affects boys, but in rare cases it can affect girls.

Using a triangular distribution of incidence rates identified in the literature2,3, a sensitivity analysis was run to estimate the diagnosed incidence of DMD in the U.S. at 17.24 per 100,000 live male births, corresponding to approximately 362 incident cases in 2019; diagnosed prevalence was found to be 6.09 per 100,000 male population across all age groups, corresponding to about 10,015 prevalent cases in 2019.

The Deerfield Institute researchers found that while the majority (64.5%) of DMD patients are under the age of 20, there is a significant number of older DMD patients up to 45 years of age that were excluded from previous prevalence estimates. 4 The prevalence of DMD among males, aged 45 or younger, was found to be 10.0 per 100,000 vs previously estimated prevalence estimates of 1.38 per 10,000 among males 5 to 24 years of age.

"We hypothesized that the prevalence of DMD has increased over the past few decades, due predominantly to improvements in treatment and care" said Emma Giegerich, MPH, an epidemiologist with the Deerfield Institute and co-author of the study. "Our incidence-to-prevalence model was built using system dynamics principles and birth-cohort-specific survival curves to get the most accurate picture of the disease landscape and its current burden. The results indicate that there is a larger than expected patient population that may benefit from novel treatment interventions, such as targeted gene therapies, potentially improving the viability of current or future drug development programs."
-end-
The study was co-authored by Mark Stuntz, MPH, a former Deerfield Institute investigator.

1 Retrieved from https://www.mda.org/disease/duchenne-muscular-dystrophy.

2 Dooley J, Gordon EK, Dodds L, MacSween J. Duchenne muscular dystrophy: a 30-year population-based incidence study. Clinical Pediatrics 2010;49(2):177-179.

3 Mendell JR, Shilling C, Leslie ND, Flanigan KM, al-Dahhak R, Gastier-Foster J, Kneile K, Dunn DM, Duval B, Aoyagi A, Hamil C, Mahmoud M, Roush K, Bird L, Rankin C, Lilly H, Street N, Chandrasekar R, Weiss RB. Evidence-based path to newborn screening for Duchenne muscular dystrophy. Ann Neurol 2012;71:304-313.

4 Romitti PA, Zhu Y, Puzhankara S, James KA, Nabukera SK, Zamba GK, Ciafaloni E, Cunniff C, Druschel CM, Mathews KD, Matthews DJ, Meaney FJ, Andrews JG, Conway KM, Fox DJ, Street N, Adams MM, Bolen J, STARnet MD. Prevalence of Duchenne and Becker muscular dystrophies in the United States. Pediatrics. 2015;135(3):513-21.

About the Deerfield Institute

The Deerfield Institute is the research division of Deerfield Management, a health care investment management firm committed to advancing health care through investment, information and philanthropy.

Deerfield Management (Deerfield Institute Division)

Related Longevity Articles:

Glucose acts as a double edged sword on longevity factor SIRT1
Aberrant fasted-to-refed transitions associated with excess glucose production and perturbed insulin signaling in the liver, are known to cause diabetes/obesity/aging.
Women, exercise and longevity
Women who can exercise vigorously are at significantly lower risk of dying from heart disease, cancer and other causes.
Deep biomarkers of aging and longevity: From research to applications
The deep age predictors can help advance aging research by establishing causal relationships in nonlinear systems.
Can plants tell us something about longevity?
The oldest living organism on Earth is a plant, Methuselah a bristlecone pine (Pinus longaeva) (pictured below) that is over 5,000 years old.
In a first, scientists pinpoint neural activity's role in human longevity
Researchers discover that the activity of the nervous system might influence human longevity.
Scientists have found longevity biomarkers
An international group of scientists studied the effects of 17 different lifespan-extending interventions on gene activity in mice and discovered genetic biomarkers of longevity.
Geneticists unlock the secret of mutant flies' longevity
Russian researchers determined which genes are affected by mutation that extends lifespan of fruit flies.
A moderate dose of novel form of stress promotes longevity
A newly described form of stress called chromatin architectural defect, or chromatin stress, triggers in cells a response that leads to a longer life.
Genetics play strong role in determining age of menopause and overall longevity
If you're wondering why you entered menopause earlier or later than other women, blame your mother.
'Longevity gene' responsible for more efficient DNA repair
University of Rochester researchers found that the gene sirtuin 6 (SIRT6) is responsible for more efficient DNA repair in species with longer lifespans.
More Longevity News and Longevity Current Events

Trending Science News

Current Coronavirus (COVID-19) News

Top Science Podcasts

We have hand picked the top science podcasts of 2020.
Now Playing: TED Radio Hour

Teaching For Better Humans 2.0
More than test scores or good grades–what do kids need for the future? This hour, TED speakers explore how to help children grow into better humans, both during and after this time of crisis. Guests include educators Richard Culatta and Liz Kleinrock, psychologist Thomas Curran, and writer Jacqueline Woodson.
Now Playing: Science for the People

#556 The Power of Friendship
It's 2020 and times are tough. Maybe some of us are learning about social distancing the hard way. Maybe we just are all a little anxious. No matter what, we could probably use a friend. But what is a friend, exactly? And why do we need them so much? This week host Bethany Brookshire speaks with Lydia Denworth, author of the new book "Friendship: The Evolution, Biology, and Extraordinary Power of Life's Fundamental Bond". This episode is hosted by Bethany Brookshire, science writer from Science News.
Now Playing: Radiolab

Space
One of the most consistent questions we get at the show is from parents who want to know which episodes are kid-friendly and which aren't. So today, we're releasing a separate feed, Radiolab for Kids. To kick it off, we're rerunning an all-time favorite episode: Space. In the 60's, space exploration was an American obsession. This hour, we chart the path from romance to increasing cynicism. We begin with Ann Druyan, widow of Carl Sagan, with a story about the Voyager expedition, true love, and a golden record that travels through space. And astrophysicist Neil de Grasse Tyson explains the Coepernican Principle, and just how insignificant we are. Support Radiolab today at Radiolab.org/donate.