• SKIP TO CONTENT
  • SKIP NAVIGATION
  • Patient Resources
    • COVID-19 Patient Resource Center
    • Clinical Trials
    • Search Clinical Trials
    • Patient Notification System
    • What is Clinical Research?
    • Volunteering for a Clinical Trial
    • Understanding Informed Consent
    • Useful Resources
    • FDA Approved Drugs
  • Professional Resources
    • Research Center Profiles
    • Clinical Trial Listings
    • Market Research
    • FDA Approved Drugs
    • Training Guides
    • Books
    • eLearning
    • Events
    • Newsletters
    • White Papers
    • SOPs
    • eCFR and Guidances
  • White Papers
  • Trial Listings
  • Advertise
  • COVID-19
  • iConnect
  • Sign In
  • Create Account
  • Sign Out
  • My Account
Home » Kurt+Peter Foundation builds online patient registry for muscular dystrophy

Kurt+Peter Foundation builds online patient registry for muscular dystrophy

December 18, 2013
CenterWatch Staff

The Kurt+Peter Foundation, a nonprofit group that supports research into potential therapies, has released the first patient registry for Limb Girdle Muscular Dystrophy, type 2C, also known as gamma sarcoglycanopathy (LGMD2C). 

Recent scientific developments suggest that new treatments for LGMD2C may be ready for testing in patients in the next few years. The patient registry will allow researchers to find patients for clinical studies.

LGMD2C is a severe disease that typically affects children between the ages of four and 12. Children with the disease lose muscle strength and typically lose the ability to walk by their teens. Medical advances, such as exon skipping, high-throughput screening of existing drugs and improved prospects for gene therapy, offer hope for treating LGMD2C.

"Recent research provides reason for optimism that clinical trials for therapies for LGMD2C are not too far in the future," said Dr. Elizabeth McNally of the University of Chicago, a LGMD2C researcher. "A registry such as the one created by the Kurt+Peter Foundation will be important for identifying and registering patients."

The LGMD2C registry is available at both www.lgmd2cregistry.org and www.kurtpeterfoundation.org/patient_registry.  It will allow patients to register to provide information that researchers will need for potential clinical trials.

"We are excited to have developed and released this registry that will allow patients to register for research for an underdiagnosed and severe disease that impacts children worldwide," said Scott Frewing, president of the Kurt+Peter Foundation. "The registry will allow clinicians to identify patients and share that information to assist clinical trials.”

Upcoming Events

  • 16Feb

    Fundamentals of FDA Inspection Management: Reduce Anxiety, Increase Inspection Success

  • 21May

    WCG MAGI Clinical Research Conference – 2023 East

Featured Products

  • Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

    Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

  • Surviving an FDA GCP Inspection

    Surviving an FDA GCP Inspection: Resources for Investigators, Sponsors, CROs and IRBs

Featured Stories

  • SurveywBlueBackground-360x240.png

    Sites Name Tech Acceptance as Essential Factor in Selection of Sponsors, Survey Finds

  • TrendsInsights2023-360x240.png

    WCG Clinical Research Trends and Insights for 2023, Part Two

  • TimeMoneyEffort-360x240.png

    Time is Money and So Is Effort, Budgeting Experts Say

  • TrendsInsights2023A-360x240.png

    WCG Clinical Research Trends and Insights for 2023, Part Three

Standard Operating Procedures for Risk-Based Monitoring of Clinical Trials

The information you need to adapt your monitoring plan to changing times.

Learn More Here
  • About Us
  • Contact Us
  • Privacy Policy
  • Do Not Sell or Share My Data

Footer Logo

300 N. Washington St., Suite 200, Falls Church, VA 22046, USA

Phone 617.948.5100 – Toll free 866.219.3440

Copyright © 2023. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing