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Muscular Dystrophy, Duchenne and Becker Types

What is Muscular Dystrophy, Duchenne and Becker Types?

Duchenne and Becker Muscular Dystrophy is a genetic condition characterized by progressive muscle weakness and atrophy which primarily affects the skeletal and heart muscles.

 

Duchenne and Becker Muscular Dystrophy is a genetic condition characterized by progressive muscle weakness and atrophy which primarily affects the skeletal and heart muscles.
Acknowledgement of Muscular Dystrophy, Duchenne and Becker Types has not been added yet.
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Synonyms for Muscular Dystrophy, Duchenne and Becker Types has not been added yet.
Mutations in the DMD gene cause Duchenne and Becker muscular dystrophy.
Symptoms for Muscular Dystrophy, Duchenne and Becker Types has not been added yet.
Diagnosis of Muscular Dystrophy, Duchenne and Becker Types has not been added yet.
Diagnostic tests of Muscular Dystrophy, Duchenne and Becker Types has not been added yet
Treatments of Muscular Dystrophy, Duchenne and Becker Types has not been added yet.
Prognosis of Muscular Dystrophy, Duchenne and Becker Types has not been added yet.
Tips or Suggestions of Muscular Dystrophy, Duchenne and Becker Types has not been added yet.
References of Muscular Dystrophy, Duchenne and Becker Types has not been added yet.
Meet the family working with scientists to fight rare diseases with CRISPR therapy Created by shannon.ashoori
Last updated 23 Jun 2020, 10:32 PM

Posted by shannon.ashoori
23 Jun 2020, 10:32 PM

Posted by shannon.ashoori
23 Jun 2020, 10:32 PM

Check out this story about Terry Horgan, a 24 year old boy, living with muscular dystrophy. Terry’s brother has founded “Cure Rare Disease”, a non profit organization which is currently exploring a treatment for muscular dystrophy. This new gene editing technique is called CRISPR. Watch the video within the provided link to learn more!

https://www.today.com/video/meet-the-family-working-with-scientists-to-fight-rare-diseases-with-crispr-therapy-85361221934

New To Rareshare Created by kxd185
Last updated 15 Feb 2010, 10:03 PM

Posted by kxd185
15 Feb 2010, 10:03 PM

I just wanted to say hi and introduce myself. My name is Kim, and I joined because of a close and personal friend of mine, Ryan Ballou. He and his father Ty started a charity called Ballou Skies to help raise awareness and funds to help fund research to prolong the lives of those with Duchenne Muscular Dystrophy. I'm interested to hear how you guys are coping with this condition and what are your hopes, dreams, aspirations and how we can get the word out about the need for research. Please check out the Ballou Skies homepage if you have time and let me know what you think. I would love to hear from you. Thanks.

Community News Articles
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LELANTOS II Duchenne Muscular Dystrophy Study

Publication date: 11 Feb 2022

Community: Muscular Dystrophy, Duchenne and Becker Types


Community External News Link
Title Date Link
Duchenne Muscular Dystrophy: Consortium.AI wants to cure rare diseases using artificial intelligence - VentureBeat 07/19/2018
Meet the family working with scientists to fight rare diseases with CRISPR therapy 06/21/2020
A Boy With Muscular Dystrophy Was Headed For A Wheelchair. Then Gene Therapy Arrived 08/02/2020
Genethon: green light from the ANSM to start a gene therapy trial for Duchenne Dystrophy 12/06/2020
Climbing Mount Everest? A courageous dad fundraises to find cure for a muscle disease in hopes of saving son’s life. 03/13/2022
Oxford-Harrington Rare Disease Centre to Advance Novel Treatment for Duchenne Muscular Dystrophy 08/07/2022
Cure Rare Disease Scores IND for First-in-Human CRISPR Therapeutic 08/14/2022
A 27-year-old with a rare disease has died in an experimental gene-editing study. He was the only volunteer. 12/03/2022
The Reasons Why I Share Our Rare Disease Story 12/24/2022
FDA advisers narrowly vote in favor of experimental gene therapy for rare muscle disease 05/14/2023
FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy 06/24/2023
We Speak Duchenne 03/01/2024
Community Resources
Title Description Date Link

Clinical Trials


Cords registry

CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access.

Enrolling is easy.

  1. Complete the screening form.
  2. Review the informed consent.
  3. Answer the permission and data sharing questions.

After these steps, the enrollment process is complete. All other questions are voluntary. However, these questions are important to patients and their families to create awareness as well as to researchers to study rare diseases. This is why we ask our participants to update their information annually or anytime changes to their information occur.

Researchers can contact CoRDS to determine if the registry contains participants with the rare disease they are researching. If the researcher determines there is a sufficient number of participants or data on the rare disease of interest within the registry, the researcher can apply for access. Upon approval from the CoRDS Scientific Advisory Board, CoRDS staff will reach out to participants on behalf of the researcher. It is then up to the participant to determine if they would like to join the study.

Visit sanfordresearch.org/CoRDS to enroll.

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Looking for information on Becker Muscular Dystrophy

my bio

My youngest adoptive son (age 13), has Duchenne Muscular Dystrophy. I also have three older children, boy 17, girl 21, and boy 26.
Pediatric Physical Therapist specializing in rare conditions and multiple disabilities
Persona en condición de discapacidad debido a distrofia muscular progresiva.

 

My husband was diagnosed with beckers muscular dystrophy about 18 months ago.
Hello my name is Rich i Was born with Myotonia Congenita or Myotonic dystrophy. I am here looking for someone in close to my area with the same thing or close. So i can have someone to share with...
Hi,

 

 

I'm joining the group because I'm involved with a local charity called Ballou Skies which was started by Ty Ballou and his quest to raise awareness of his son Ryan's condition of...
Married 30 years, mother of 2 young men. One is 27 and the other 14 years young. Careers previously centered around breeding, training and schooling horses and training students for high level...
Is 9 years old with DMD.
I work as an intern for the Genetic Alliance, an advocacy organization for individuals and groups with rare genetic conditions. The Genetic Alliance provides many resources with information about...

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New To Rareshare

Created by kxd185 | Last updated 15 Feb 2010, 10:03 PM


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