Hi folks, if you can, please donate today and help Dec smash his target and fulfil his bucket list.
Thank you so much. 🙏
#HelpDec #DecsBucketList #DuchenneMuscularDystrophy

gofund.me/a63da1cb

On May 12, 2023, independent experts narrowly recommended that the FDA grant accelerated approval to Sarepta Therapeutics's new gene therapy, SRP-9001 (delandistrogene moxeparvovec), for the treatment of Duchenne muscular dystrophy (DMD)
—Reshma Ramachandran and Holly Fernandez Lynch
medpagetoday.com/opinion/second…

ojrd.biomedcentral.com/articles/10.11…

Feeling blessed to support some children in Punjab & Haryana suffering from Duchenne muscular dystrophy (DMD) which can't be cured permanently but needs regulat medication & families are not able to afford such expensive medicines.
#SevaForAll #worktogether

There were no dose-limiting toxicities, serious adverse events, nor infusion-related reactions among 6 patients treated with ENCell’s investigational EN001 therapy.

ow.ly/HAiX50OzY0x

mdpi.com/2076-3921/9/12…

Try 1 roll of custom packaging tape for $9.

Ten years ago today, Max was first diagnosed with Duchenne muscular dystrophy. It was news that shattered our world and changed our lives forever. At first, it was impossible to sleep because of the terrible thoughts running through our minds – so we listened to podcasts in bed..

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Halli My son has Duchenne Muscular Dystrophy. So I know what you are going through.

Cornell University graduates, brothers on forefront of Duchenne muscular dystrophy cure IthacaJournal
ithacajournal.com/picture-galler…

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FDA delays decision on Sarepta’s gene therapy for Duchenne statnews.com/2023/05/24/fda…

Excited to welcome back our June #SaveToSupport cause Jett Foundation. If we save people over $10,000 this month (as we've done 106 months in a row) we'll donate $1,000 to their important work on behalf of kids with Duchenne Muscular Dystrophy.

Check out our most recent blog post!

ibl-america.com/blog/publicati…

#SportsMedicine #MuscularDystrophy #NeuromuscularDisorders

'Duchenne muscular dystrophy is a genetic disorder caused by mutations in the dystrophin gene, leading to the absence or reduction of the dystrophin protein.

The investigational gene therapy, also known as SRP-9001, was recently backed by the agency’s advisory committee in a tight decision. Its new deadline for review is June 22, 2023.

ow.ly/26QK50Owa55

In 2021, Pfizer reported the death of a patient in its early-stage Duchenne muscular dystrophy gene trial. And in a major earlier setback for the gene therapy field, 18-year-old Jesse Gelsinger died in 1999 during a study to combat a rare metabolic disease
nbc-2.com/news/2022/11/0…

The #FDA is delaying by one month a decision on the approval of a gene therapy for Duchenne muscular dystrophy, the treatment’s maker, Sarepta Therapeutics.

Is this the right call?

$SRPT #genetherapy #duchenne #RareDisease

There were 18 AEs observed among the group treated with ENCell’s EN001; the AEs were deemed mild, and the patients recovered from them within 5 days of onset.

#ASGCT23

Read more:

ow.ly/7Bsm50OyzOo

ncbi.nlm.nih.gov/pmc/articles/P…

Sarepta Therapeutics said the FDA is working toward potentially granting an accelerated approval for SRP-9001 but only for 4- and 5-year-old patients. The company had asked the agency to give the drug accelerated approval for all Duchenne patients who could still walk
statnews.com/2023/05/24/fda…

Thank you Cure Rare Disease and Rich Horgan for publishing this — this must have been so tough to write. Open sharing, even with an unexpected outcome, is critical to learning & moving rare disease treatments forward. Terry's legacy lives on 🙏 medrxiv.org/content/10.110…