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COMMERCIAL-IN-CONFIDENCE (After first entry) Commonwealth of Australia Department of Defence APPLICATION TO EXPORT CONTROLLED GOODS Please refer to the Instruction Sheet when completing this form.

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The latest, significant research finding, published in Human Molecular Genetics, February 2022, involves the small-molecule which has been shown to mitigate DMD-related skeletal muscle disease in a number of ways.

The prognosis for patients with Duchenne muscular dystrophy is bleak. Most use a wheelchair by the time they are teenagers and don't typically live into their 30s. There is no cure for the disease.

The team is developing a new technology called “Cure Duchenne Muscular Dystrophy (Cure DMD).” The hope is to treat DMD before birth by editing the genes that encode, or cause the production of, dystrophin.

Duchenne muscular dystrophy is caused by a defective gene for dystrophin (a protein in the muscles). However, it often occurs in people without a known family history of the condition. Duchenne muscular dystrophy occurs in about 1 out of every 3,600 male infants.

Duchenne MD usually becomes apparent during the toddler years, sometimes soon after an affected child begins to walk. Progressive weakness and muscle wasting (a decrease in muscle strength and size) caused by degenerating muscle fibers begins in the upper legs and pelvis before spreading into the upper arms.

Currently, there is no non-viral gene editing treatment that can fully correct the mutation of the DMD gene or restore full-length dystrophin before birth.

Patients with DMD typically lose their ability to walk in their teenage years and develop heart and lung complications as they age. Treatments for DMD are limited and there is no known cure.

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© Copyright 1997-2025
airSlate Legal Forms, Inc.
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Form Packages
Adoption
Bankruptcy
Contractors
Divorce
Home Sales
Employment
Identity Theft
Incorporation
Landlord Tenant
Living Trust
Name Change
Personal Planning
Small Business
Wills & Estates
Packages A-Z
Form Categories
Affidavits
Bankruptcy
Bill of Sale
Corporate - LLC
Divorce
Employment
Identity Theft
Internet Technology
Landlord Tenant
Living Wills
Name Change
Power of Attorney
Real Estate
Small Estates
Wills
All Forms
Forms A-Z
Form Library
Customer Service
Terms of Service
DMCA Policy
About Us
Blog
Affiliates
Contact Us
Privacy Notice
Delete My Account
Site Map
All Forms
Search all Forms
Industries
Forms in Spanish
Localized Forms
Legal Guides
Real Estate Handbook
All Guides
Prepared for You
Notarize
Incorporation services
Our Customers
For Consumers
For Small Business
For Attorneys
Our Sites
US Legal Forms
USLegal
FormsPass
pdfFiller
signNow
airSlate workflows
DocHub
Instapage
Social Media
Call us now toll free:
1-877-389-0141
As seen in:
  • USA Today logo picture
  • CBC News logo picture
  • LA Times logo picture
  • The Washington Post logo picture
  • AP logo picture
  • Forbes logo picture
© Copyright 1997-2025
airSlate Legal Forms, Inc.
3720 Flowood Dr, Flowood, Mississippi 39232