MJ and Duchenne

Build Your Own Care Binder at PPMD’s 30th Annual Conference PPMD is excited to share that at our 30th Annual Conference, taking place June 27-29, 2024, in Orlando, Florida, the PPMD Care team will host a “DIY Care Binder” station where families can create a...

MJ is Approved for the MDCP!! Thanking God for this blessing! 👏👏🙏🙏

Get ready for some June basketball, MFFLs.

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Enter our raffle for a chance to win 50lbs of premium beef!
Tickets are just $5 each, with eight boxes of beef per drawing. The raffle will be drawn on September 19, 2024, at BLS in Billings, Montana. Winners will be notified and do not need to be present.

Hosted by Paul and Laura Heaton, this event supports CureDuchenne's mission to find a cure for Duchenne muscular dystrophy.

Join us in accelerating research, improving care, and empowering the Duchenne community!
🎟️ Buy raffle tickets here: https://e.givesmart.com/events/C3k/
Calves to cure DMD

2024 Abilene Camp Rehab Day 1 - WestTexasRehab This gallery hosted by SmugMug; your photos look better here.

Day 2 at Camp Rehab was a success! One more day! Mj’s buddy won’t be there today as he has to go to football practice. Thankful for all the buddies that dedicate their time to the kiddos. Camp Rehab couldn’t happen without you all!

Day 1 of Camp was a success for MJ! We love West Texas Rehab Center and Camp Rehab!! Always a grand time and he makes so many awesome memories. His buddies are always perfect for him thanks to his PT/OT! 🫶🏼

Praying for this family 😢🙏

We at CureDuchenne are extremely saddened to learn that a young patient in Pfizer’s Phase 2 DAYLIGHT study (for boys 2 years to less than 4 years of age) has suddenly passed away. The young man received the investigational micro-dystrophin gene therapy, fordadistrogene movaparvovec, over one year ago in early 2023.

While the cause of death is currently unknown, Pfizer is committed to sharing more information as it becomes available, and our hearts break for the family and friends of this young boy at this difficult time.

“We are devasted to learn of this heartbreaking loss and our hearts are profoundly with the boy’s family during this unimaginably difficult time. This tragic event painfully reminds us of the stakes of our mission and the importance of ensuring the utmost safety in all clinical trials,” said Debra Miller, founder and CEO of CureDuchenne. “We recognize the tremendous courage of this family – and all families that participate in clinical trials - as we offer the support of the whole Duchenne community.”

We are forever grateful to the broader Duchenne community for their critical participation in clinical trials and recognize that substantial advances to treatment require Biopharma/community partnerships to drive innovation. Together, we reaffirm our commitment to safety, transparency, and progress in Duchenne research and will work tirelessly to support families every step of the way in their journey.

Read our blog and the community letter here:
https://cureduchenne.org/research/update-on-pfizers-daylight-gene-therapy-trial-for-duchenne/

EDGEWISE THERAPEUTICS ANNOUNCES POSITIVE TWO-YEAR TOPLINE RESULTS Edgewise Therapeutics Announces Positive Two-Year Topline Results from the ARCH Open Label Trial of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy (Becker)

Have you got your social media pledge cards ready? 📝🌍

Available to download from our website, our pledge cards are written in 8 languages, allowing you to show your passion for Rare Disease Day, in your local contexts!

Download them today alongside our other resources by clicking on our website here: https://www.rarediseaseday.org/downloads/. Show your support for the international rare disease community today! 💻💡

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Yay! The new all-abilities playground is done! Glad we were able to make the ribbon cutting! Thank you City of Sweetwater

Register for this year's Walk For Wishes El Paso 🏃‍♀️! Join us for a family-friendly event and help us raise funds to grant wishes for local children here in El Paso. Secure your spot before prices increase. 👉https://ow.ly/sGUa50QJ0xj

I LOVE THIS ANALOGY:
You are holding a cup of coffee when someone comes along and bumps into you or shakes your arm, making you spill your coffee everywhere.
Why did you spill the coffee?
"Because someone bumped into me!!!"
Wrong answer.
You spilled the coffee because there was coffee in your cup.
Had there been tea in the cup, you would have spilled tea.
Whatever is inside the cup is what will spill out.
Therefore, when life comes along and shakes you (which WILL happen), whatever is inside you will come out. It's easy to fake it, until you get rattled.
So we have to ask ourselves... “what's in my cup?"

Joshua really struggles with positive spills at the minute and constant asks when do I remember him geing happy 😪 I asked him the same question and he replied
"Just before lockdown when I was in year 6". That's 4 years ago. So much has happened within our family since that time that's been difficult to deal with and we need to make a spill shift.

When life gets tough, what spills over?
Joy, gratitude, peace and humility?
Anger, bitterness, victim mentality and quitting tendencies?
Life provides the cup, YOU choose how to fill it.
Today let's work towards filling our cups with gratitude, forgiveness, joy, words of affirmation, resilience, positivity; and kindness, gentleness and love for others.

Yesterday we took a trip to pick up something amazing! A new power chair for MJ! It’s perfect for him and was generously given to him by Sam. The Killian family is beautiful and blessed our boy tremendously. His independence at school will flourish and he’ll be able to keep up with his friends.

We are so grateful 🥹🥲💚

Once MJ gets home from grandmas and papas house we have something exciting to share with y’all!!! 💚🙏🥹

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This place does AMAZING things and we are so glad MJ has weekly pt/ot here!

🎉 $1,747,238 Raised to support the Rehab! 🎉

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"Maybe, just maybe, if you could find a way to bring some joy and hope to those with Duchenne, my heart could grow three sizes with gratitude." What The Grinch might say....
The Grinch UK

Thank you Jason's journey with Duchenne for making this for us. 😁😁

Counting down until my Wish trip! Thank you Make a Wish North Texas

Today we received our flight itinerary for Mj's wish from Make-A-Wish North Texas ! He is pumped and said come on January 14th!