Research (1,236)

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Propose research that is important to you

Adding your experiences with Duchenne or Becker to ongoing research is vital to improve care and optimize potential therapies. But our com…

Started by Holly PeayLatest Reply


Staff

If you had a chance to ask researchers, providers or industry any question at all about clinical trials, what would it be?

If you had a chance to ask researchers, providers or industry any question at all about clinical trials, what would it be? PPMD’s 2012 Co…

Started by Kathi KinnettLatest Reply

Heart Transplant update

We wanted to give a update on Josh. Joshs 3yr. Heart Transplant anniversary is March 24!!! Transplant CAN be done in DMD!!!! Josh is prepar…

Started by TammyLatest Reply

Discussions Replies Latest Activity

New CRISPR-EXON SKIP technique could treat DMD

https://www.sciencedaily.com/releases/2018/08/180816153147.htm "By editing a single base in genomic DNA using CRISPR-SKIP, we can eliminate…

Started by Jason Darienzo

0 Aug 17

Muscular dystrophy patients could someday also benefit from this hydrogel successfully tested in mice

https://www.sciencedaily.com/releases/2018/08/180815154502.htm Muscles of the elderly and of patients with Duchene muscular dystrophy have…

Started by Jason Darienzo

0 Aug 17

Heat therapy boosts mitochondrial function in muscles

Could help with hand function?. PPMD should look at this.  Heat therapy boosts mitochondrial function in muscles

Started by Jason Darienzo

0 Jul 31

Summit Announces PhaseOut DMD Did Not Meet Primary Endpoint

Summit Announces PhaseOut DMD Did Not Meet Primary EndpointEzutromid Development to be DiscontinuedSummit to Focus on Advancing its Pipelin…

Started by Jason Darienzo

1 Jul 11
Reply by Jason Darienzo

Steriod use at a later age?

Hello, My son just turned 14 yrs old and was diagnosed with DMD at age 3. At the time of his diagnosis both his doctor and us agreed not to…

Started by Rahel kebede

0 Jun 20

Utrophin

Does anyone know if Summit is planning on conducting a Phase 3 trial after the Phase 2 trial is successful and we get positive results in t…

Started by Sampreet

1 May 31
Reply by Aleksandar

Exon 52 deleted

What medication do you use. Exondys 51?

Started by Apple

0 May 26

Cholesterol

Hi Have any of your boys experienced higher cholesterol and if so, have they been given statins or any other medication to address this? 

Started by Marks33

0 Apr 27

New CRISPR method efficiently corrects Duchenne muscular dystrophy defect in heart tissue

Scientists have developed a CRISPR gene-editing technique that can potentially correct a majority of the 3,000 mutations that cause Duchenn…

Started by Jason Darienzo

1 Feb 7
Reply by Galia Ben Ishay

low back pain

does anyone here know what should i do with my kid as he suffers from chronic low back pain. i mean is there any treatment that we should d…

Started by ahmed Sayed Abdel Gawad

1 Feb 5
Reply by Kathi Kinnett

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