Wednesday, September 27, 2023
OANDP-L
  • Login
No Result
View All Result
The O&P EDGE
  • PECOS
  • Magazine
    • Subscription
    • Current Issue
    • Issue Archive
    • News Archive
    • Product & Service Directory
    • Advertising Information
    • EDGE Flipbooks
  • O&P Jobs
    • Find a Job
    • Post a Job
  • EDGE Advantage
    • EA Homepage
    • EA Data
  • O&P Facilities
  • Resources
    • Product & Service Directory
    • Calendar
    • Contact
    • About Us
    • O&P Library
    • The Guide
    • Custom Publications
    • Advertising Information
    • EDGE Direct
    • Amplitude Media Group
  • PECOS
  • Magazine
    • Subscription
    • Current Issue
    • Issue Archive
    • News Archive
    • Product & Service Directory
    • Advertising Information
    • EDGE Flipbooks
  • O&P Jobs
    • Find a Job
    • Post a Job
  • EDGE Advantage
    • EA Homepage
    • EA Data
  • O&P Facilities
  • Resources
    • Product & Service Directory
    • Calendar
    • Contact
    • About Us
    • O&P Library
    • The Guide
    • Custom Publications
    • Advertising Information
    • EDGE Direct
    • Amplitude Media Group
No Result
View All Result
The O&P EDGE Magazine
No Result
View All Result
Home News

Duchenne Muscular Dystrophy: Research Takes Personal Turn for UCLA Scientists

by Miki Fairley
January 2, 2022
in News
0
SHARES
16
VIEWS
Share on FacebookShare on Twitter

Stanley Nelson, MD, is a professor in the Department of Human Genetics at the University of California, Los Angeles (UCLA); his wife, Carrie Miceli, PhD, is a professor at the UCLA Department of Microbiology, Immunology, and Molecular Genetics. Both are researchers in the quest to find better therapies and even a possible cure for Duchenne muscular dystrophy (DMD), the most frequent and most lethal form of muscular dystrophy (MD). They are also parents to two boys, Calvin, 18, who entered college this year, and Dylan, 11, who was born with DMD.

While Dylan entered the world carrying a heavy genetic burden, he has two powerful forces going for him: his loving, caring parents who have the knowledge and skills to fight to find better therapies and solutions.

In 2008, the husband-and-wife team made a huge stride in their professional and personal efforts in the search for new treatments for DMD. With funding assistance from several private and government organizations, they opened the Center for Duchenne Muscular Dystrophy at UCLA (CDMD@UCLA) to support clinical care and translational research. Along with Melissa Spencer, PhD, a professor of neurology at UCLA, they serve as the center’s directors.

CDMD@UCLA investigators are involved in several new research areas, among them, exon-skipping therapy. In DMD, lack of the protein dystrophin causes muscle deterioration and breakdown, leading to the devastating effects of the disease. Exon skipping has been likened to a “band-aid” to skip over the parts of the gene that block the effective creation of this essential protein.

Among other projects, center investigators are searching for drugs to enhance the effectiveness of exon skipping, using such techniques as high throughput screening (HTS) and repurposing drugs already approved by the Food and Drug Administration (FDA) for other conditions, but which also show promise for DMD therapy. HTS technologies enable researchers to rapidly identify active compounds, antibodies, or genes that modulate a particular biomolecular pathway, thus providing starting points for drug design, among other uses.
Exon-skipping drug therapies are currently in human clinical trials, Nelson says.

Dylan’s Role

Dylan, too, has played a part in various clinical trials. “He feels good about helping other boys with Duchenne muscular dystrophy,” Miceli says. He has participated in a natural history study tracing progression of the disease and is currently participating in a drug-therapy study in cooperation with Cedars-Sinai Medical Center, Los Angeles, to determine if Viagra® and related drugs can improve muscle blood flow in DMD patients. Improved blood flow is predicted to lessen the damage to contracting skeletal muscles, thus reducing and slowing muscle damage and deterioration.

“Dylan is also traveling to Florida to participate in an imaging study,” Nelson says. “This study examines whether or not using imaging instead of function tests will give us a more accurate picture of how the disease is progressing and what effect therapies are having. Dylan also gets to go to Disney World, which he’s excited about.”

At age 11, Dylan’s muscles are weakening more. He no longer can participate in sports, has difficulty with stairs, and uses a scooter for some activities, such as field trips. Current medical care includes corticosteroids, angiotensin-converting-enzyme (ACE) inhibitors to slow development of cardiomyopathy, and nutritional supplements. Dylan uses night-time orthoses to keep his Achilles tendon stretched to avoid contractures and alleviate toe walking, and has a daily stretching routine.

Miceli strongly urges parents to enlist the help of a multidisciplinary team. “The literature is quite clear that multidisciplinary care can add 10-15 years to life expectancy.” She also urges parents not to accept the fatalistic view of some physicians that their son likely will only live into his teens or early 20s. “With good care and a multidisplinary team, life expectancy is being pushed up into the 20s and 30s [with some patients living into their 40s]. And promising clinical trials are coming down the pike.”

As for Dylan, in spite of the debilitating effects of DMD, “He is a happy, cheerful boy,” Miceli says. Dylan—and his parents—are glad that all their efforts may not only help him, but also all the other boys with DMD.


Related posts:

  1. Duchenne Muscular Dystrophy
    The Search for New Treatments Races against the Clock
  2. Health-related Quality of Life: A Developing Standard in Healthcare
  3. The Changing Landscape: Manufacturers Enter the US Clinical Care Market
  4. The Missing Link: How Dialogue Can Transform Hip Dysplasia Care
Tags: NULL
Previous Post

NAAOP Releases Webcast, Issues Action Alert about H.R. 4175, Insurance Fairness for Amputees Act

Next Post

Documentary Chronicles Life of Paul E. Leimkuehler

Next Post

Documentary Chronicles Life of Paul E. Leimkuehler

  • VIEW CURRENT ISSUE
  • SUBSCRIBE FOR FREE

RECENT NEWS

News

Smaller Inverted Orthosis Angle Beneficial for Women With Flexible Flatfeet

by The O&P EDGE
September 22, 2023

A research team examined the effects of different inverted angles of foot orthoses on walking kinematics in women with flexible...

Read more

Gait Differs With Lower-limb Devices: A Literature Review

Women With Limb Loss Cite Prosthetist Relationships in Needs Survey

Ottobock Opens Production Center in Bulgaria

Get unlimited access!

Join EDGE ADVANTAGE and unlock The O&P EDGE's vast library of archived content.
SUBSCRIBE TODAY

O&P JOBS

Eastern

Full Time Certified Ped Orthotist

Eastern

CO/Certified Orthotist

Eastern

Customer Service Representative

 

© 2023 The O&P EDGE

  • About
  • Advertise
  • Contact
  • EDGE Advantage
  • OANDP-L
  • Subscribe

CONTACT US

866-613-0257

[email protected]

201 E. 4th St
Loveland, CO 80537

The most important industry news and events delivered directly to your inbox every week.

  • About
  • Advertise
  • Contact
  • EDGE Advantage
  • OANDP-L
  • Subscribe

© 2023 The O&P EDGE

No Result
View All Result
  • PECOS
  • MAGAZINE
    • SUBSCRIBE
    • CURRENT ISSUE
    • ISSUE ARCHIVE
    • NEWS ARCHIVE
    • PRODUCTS & SERVICES DIRECTORY
    • ADVERTISING INFORMATION
  • O&P JOBS
    • FIND A JOB
    • POST A JOB
  • EDGE ADVANTAGE
    • EA Homepage
    • EA Data
  • FACILITIES
  • RESOURCES
    • PRODUCTS & SERVICES DIRECTORY
    • CALENDAR
    • CONTACT
    • ABOUT US
    • O&P LIBRARY
    • THE GUIDE
    • CUSTOM PUBLICATIONS
    • ADVERTISING
    • EDGE DIRECT
    • AMPLITUDE
  • OANDP-L
  • LOGIN

© 2023 The O&P EDGE

Welcome Back!

Login to your account below

Forgotten Password?

Retrieve your password

Please enter your username or email address to reset your password.

Log In
The O&P EDGE Magazine
 
Required 'Candidate' login to applying this job. Click here to logout And try again
 

Login to your account

  • Forgot Password? | Sign Up

Reset Password

  • Already have an account? Login

Enter the username or e-mail you used in your profile. A password reset link will be sent to you by email.

Signup to your Account

  • By clicking checkbox, you agree to our Terms and Conditions and Privacy Policy

    Already have an account? Login

Close
Are you sure want to unlock this post?
Unlock left : 0
Are you sure want to cancel subscription?
 

Account Activation

Before you can login, you must activate your account with the code sent to your email address. If you did not receive this email, please check your junk/spam folder. Click here to resend the activation email. If you entered an incorrect email address, you will need to re-register with the correct email address.