Donate

Exon - Cure Duchenne Association

we'll find cure for Duchenne

What is Duchenne (DMD) ?

Duchenne Muscular Dystrophy 

Is the most common and severe genetic disorder affecting children, especially males. Children in Duchenne suffer from various defects in the gene responsible for the production of dystrophin protein, which is essential for muscle cell building. As a result of impaired muscular dystrophin supply, the muscle structure is impaired so that weakened muscle cells suffer from a significant decrease in function and cause a gradual loss of motor capabilities. Initial signs of illness will usually appear around age 3, leading the sick child to wheelchair in the early years of his second decade. Most patients do not survive until the age of 30 due to heart or lung dysfunction.

DNA
1
initial signs age
1
In israel
0
1 in every 3,500 live male births
1
worldwide
1
WORLWIDE
1
In ISRAEL
1
Initial signs age
0
1 in every 3,500 live male births

About us

The association was founded by the parents of a sick boy. After an early diagnosis of the disease at the age of 1.5, the parents, with the help of their family and close friends, began to research the disease and the state of the medical research related to it. The strong desire to influence the child's fate in every possible way and to give him and many like him a better chance to live normal life led the parents to initiate the founding of the association.

What we are doing

The association operates in Israel and other countries around the world and focuses on seeking and initiating innovative medical treatments and research to find a generic drug that will be a life-saving treatment for hundreds of thousands of children worldwide, regardless of the type of genetic disorder. As for today (2020), there are a number of initial drugs to treat Duchenne and Beker, these drugs have recently been granted by accelerated approval for commercial use by the American FDA, but these drugs address only a small percentage of Duchenne's children. This is due to the fact that the existing drugs provide a solution for only a limited number of genetic defects among a variety of hundreds of different defects that cause Duchenne & Beker

Starategy

Startup mode of work

Using lean startup methodologies to increase time to solutions with maximum efficiency

Data Analytics & AI

Collecting anonymous data about Duchenne and analyze it to find treatment in new ways

Medical Research

Initiation and funding of new medical research, cooperate to fund and support existing research

Full financial transparency

As a nonprofit organization which has founded with one goal - to find a cure for Duchenne, full financial transparency is a key value of the organization to its stakeholders, families, volunteers, and donors. We believe this is a prerequisite for the existence and success of the association to find the cure as soon as possible

Medical Research

Clinical Research & Trials

Clinical trials evaluate the effectiveness and safety of medications or medical devices by monitoring their effects on large groups of people.

Clinical research trials may be conducted by government health agencies such as NIH, researchers affiliated with a hospital or university medical program, independent researchers, or private industry

How to diagnose

A diagnosis of DMD is made based upon a thorough clinical evaluation, a detailed patient history, and a variety of specialized tests including molecular genetic tests. If the genetic tests are not informative, surgical removal and microscopic examination (biopsy) of affected muscle tissue that may reveal characteristic changes to muscle fibers. Specialized blood tests (e.g. creatine kinase) that evaluate the presence and levels of certain proteins in muscle (immunohistochemistry) are also used.

Signs & Symptoms

In children with DMD, initial findings may include delays in reaching developmental milestones such as sitting or standing without assistance; toe walking; an unusual, waddling manner of walking (gait); difficulty climbing stairs or rising from a sitting position (Gower’s sign); and repeated falling. Toddlers and young children may seem awkward and clumsy and may exhibit abnormal enlargement of the calves due to scarring of muscles (pseudohypertrophy).

Our Partners

Contact Us

Families, donors and volunteers
Join us to find cure for Duchenne & Beker

Email: Info@exondmd.org
Facebook-f
Linkedin

© 2020 All rights reserved exondmd.org

×