CureDuchenne

Get your raffle tickets and/or sponsor today!!
https://cureduchenne.org/calvestocure/

While we are an all class cattle sale we also have ways you can help us raise money to fund Duchenne Muscular Dystrophy research if you don’t have an animal to sell.
You can go to:
www.cureduchenne.org/calvestocure and buy raffle tickets from our online raffle. Tickets are $5 apiece and enter you into a chance to win one of eight boxes of beef. We will draw for winners sale day (Sept 19) and beef will be delivered to your house anywhere in the lower 48 states the beginning of January.
Laura and I provided the steer this year and Nick and JayDee Lindeen (Grants aunt and uncle) are in the process of feeding him out right now. Processing has once again been donated by Cowboy Meat Co. of Forsyth MT whith shipping handled by Billings Seafood Guys and paid for by Stockman Bank. Thank you and “Eat Beef CureDuchenne” see you at the sale. Please share.








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Thank you to the community of Madison, WI, for an incredible Caregiver Dinner session! 💙 💙 💙

Your presence and participation made the evening truly special. Together, we’re making strides towards better care and support for all affected by Duchenne.
https://cureduchenne.org/events/list/

Big thanks to the Tercano family for sharing their story and to Orlando magazine for raising awareness about Duchenne Muscular Dystrophy.
💙 Read their inspiring journey below:

Local Honor Roll Student & Family Share About Duchenne Muscular Dystrophy - Orlando Magazine A personal look at a Kissimmee family battling the disease.

💙 Exciting News! 💙
CureDuchenne celebrates the 's launch of the Rare Disease Innovation Hub, a groundbreaking initiative to accelerate therapy development for rare diseases like Duchenne and Becker muscular dystrophy.

This Hub will centralize engagement, enhance collaboration, and advance regulatory science to bring hope and effective treatments to our community. Stay tuned for updates as we work together for a brighter future!

FDA Rare Disease Innovation Hub to Advance Outcomes for Patients The FDA plans to establish a Rare Disease Innovation Hub. Our vision and goal for the Hub is ultimately to improve outcomes for patients.

🎧 On this powerful episode of the "In Sickness" podcast, dad Josh Argall opens up about the emotional rollercoaster following his son Devin's diagnosis with Duchenne muscular dystrophy.

From misdiagnoses to finding hope, Josh shares his journey and determination to fight for his son's future. Special guest Debra Miller, founder and CEO of CureDuchenne, provides insights into the disease and the caregiver's journey. Don't miss this inspiring story of resilience and hope. Listen now: https://bit.ly/InSickPod

Or listen on:
Apple: https://apple.co/3Y9Ynok

Spotify: https://spoti.fi/3WruOgv

Amazon Music: https://bit.ly/amazonPODsick

iHeart: https://ihr.fm/3YqUVWz

Had a great meeting with Noga Leviner and Dan Drozd, MD MSc from PicnicHealth, a valued partner of . Did you know that the average Duchenne patient sees over 20 unique healthcare providers per year? When it comes to managing care for a loved one, it can be overwhelming. Trust me, been there.

When you participate in , our data-integrated that’s open to individuals with , , and , you are not only helping advance but you also get free access to your complete medical records in one simple online portal through – a true lifesaver when it comes to managing a complex disease like Duchenne.

Learn more and how to participate
https://cureduchennelink.org/

You’re invited to “A Night of Hope” in San Antonio, TX!

Dealing for is an event that brings togetherthe San Antonio community with a global networkof families, caregivers, and compassionate individuals like yourself. Together, we will make a difference in finding a cure for Duchenne.



Learn more and get tickets:
https://dealingforduchenne.org/

Los invitamos a escuchar el podcast de Akari foundation en colaboración con duchennetijuana ♿️💚

https://open.spotify.com/episode/289HIblFmoaZxPuPjHoAE1?si=0fPsZ4evSyW3EOzj9adxrg&t=4161

Hidden Truths: Social Exclusion Thank you to psychologist Dr. Natalie Truba (of Nationwide Children’s Hospital) for identifying these “elephants in the room” in our webinar Understanding BDMD Behaviors for the Adolescents and Adu…

NeurologyLive recently chatted with Debra Miller and Dr. Michael Kelly to discuss our recent webinar on ELEVIDYS, the newly approved gene therapy for Duchenne muscular dystrophy. Watch the full interview to hear their insights on ELEVIDYS, the future of gene therapy, and our ongoing efforts to improve treatments.

You can also watch the full ELEVIDYS webinar here: https://www.youtube.com/watch?v=NPdHtwMYdcY&t=2421s

Sarepta Therapeutics

Broad FDA Approval of Gene Therapy Expands Treatment Choices for Duchenne Muscular Dystrophy: Debra Miller & Michael Kelly, PhD The chief executive officer and the chief scientific officer at CureDuchenne talked about a recent webinar that discussed the broad FDA approval of a gene therapy for Duchenne muscular dystrophy, highlighting patient choice and the ongoing efforts to improve treatments. [WATCH TIME: 9 minutes]

🎆 Happy Independence Day from CureDuchenne! 🇺🇸

We celebrate , , and . As we enjoy this special day, let’s also remember our mission to find a cure for muscular dystrophy. Your support makes a difference every day. Together, we are will cure Duchenne. 💙

Join us for a full-day event focused on improving the quality of life for individuals living with Duchenne.

PRESENTATIONS INCLUDE: Standards of Care | Family Support | Physical Therapy & Medical Equipment | Research & Clinical Trials | Open House. Breakfast & lunch are provided.

Venue: The Westin Minneapolis
Address: 88 South 6th Street, Minneapolis, Minnesota 55402

REGISTER HERE
https://web.cvent.com/event/5f182fab-a00b-483f-a9b3-b190647b9f8f/summary

REMINDER:: WEBINAR WITH DYNE THERAPEUTICS | Dyne will share with the community recent efficacy and safety data from the ongoing DELIVER trial of DYNE-251 in Duchenne.
Wednesday, July 3, 2024
9:00 AM - 10:00 AM PST

Dyne Therapeutics: Efficacy and safety data from the ongoing DELIVER trial of DYNE-251 in Duchenne Dyne Therapeutics will share with the community recent efficacy and safety data from the ongoing DELIVER trial of DYNE-251 in Duchenne.

Nathan, a talented student from The University of Texas at Austin, has been accepted into the Austrian American Mozart Academy in Salzburg! Despite the challenges of living with Duchenne muscular dystrophy, Nathan has secured a leading role in an opera during the academy. This opportunity is a dream come true!

However, his travel and accommodations are costly due to his care needs. He's raising $15,000 to help make this journey. Every bit helps! Let’s support Nathan in achieving his dreams. All funds go directly to Nathan.

Please help Nathan: https://fundraise.givesmart.com/e/7QKUMA?vid=16acg6

Join Us for FUTURES National Family Conference 2025 in San Antonio, Texas – A Transformative Event for the Duchenne Community!

Registration is now open:
https://web.cvent.com/event/3d756dbc-811a-4700-954e-ef31538e1a21/summary

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Thank you, DMD (Duchenne Muscular Dystrophy) News for this valuable resource on physical therapy in Duchenne muscular dystrophy featuring insights from our very own Physical Therapist, Jennifer Wallace PT Read more here:

Duchenne Physical Therapy

Duchenne muscular dystrophy: Exercise and physical therapy –... Exercise and physical therapy can help a person with Duchenne muscular dystrophy maintain mobility, flexibility, and independence.

Seminario Web Cure Duchenne: Explicación de la Decisión de la FDA acerca de ELEVIDYS

Seminario Web Cure Duchenne: Explicación de la Decisión de la FDA acerca de Elevidys

WEBINAR:: The FDA Approval of Sarepta Therapeutics's ELEVIDYS to Duchenne Patients Ages 4 and up.

WATCH WEBINAR BELOW:
https://www.youtube.com/watch?v=NPdHtwMYdcY

FDA Decision on Sarepta's ELEVIDYS Webinar This webinar discusses a groundbreaking gene therapy treatment for Duchenne Muscular Dystrophy (DMD), focusing on a method to introduce a miniaturized versio...

REMINDER TO REGISTER TO ATTEND! Starting soon!!

FDA Grants Traditional Approval for Ambulatory Individuals and Accelerated Approval for Non-Ambulatory Individuals to Sarepta Therapeutics’ Elevidys Gene Therapy for Duchenne Muscular Dystrophy.

We’re pleased to share that Sarepta Therapeutics' gene therapy for Duchenne, Elevidys, has been granted traditional approval for ambulatory individuals and accelerated approval for non-ambulatory individuals by the FDA for individuals with Duchenne with a confirmed mutation in the DMD gene who are at least 4 years of age.

First approved by the FDA under the accelerated approval pathway for boys ages 4-5 with Duchenne in June 2023, it’s the first approved gene therapy for Duchenne.
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Join for a webinar unpacking the recent decision on Therapeutics’ , and what it means for the community. Hear from Neurology and Neuromuscular Care Center - Diana Castro MD a board-certified neurologist and leading neuromuscular physician, who will share the latest information and her perspective as a physician treating patients with this gene therapy.

A representative from Sarepta Therapeutics will also join by invitation as a guest for a Q&A session.

Register:
https://us02web.zoom.us/webinar/register/WN_gKgCTHOpQ82JDqwyJMC27A #/registration
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Please read our blog here: https://cureduchenne.org/research/sarepta-therapeutics-announces-expanded-us-fda-approval-of-elevidys-to-duchenne-muscular-dystrophy-patients-ages-4-and-above/

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Genethon reported positive results from its AAV-microdystrophin treatment trial in Europe. The three participants receiving the study’s higher dose (3x1013vg/kg) showed that an average of 54% of muscle fibers were producing microdystrophin, and individuals demonstrated a reduced level of creatine kinase, a marker of muscle damage. Genethon said it is planning a pivotal trial in Europe.

Genethon’s Duchenne Muscular Dystrophy Gene Therapy GNT0004 Produces Expression of Microdystrophin in Phase 1/2/3 Trial Genethon’s GNT0004, an investigational recombinant adeno-associated virus (AAV) vector-based gene therapy intended to treated Duchenne muscular dystrophy (DMD), has demonstrated the ability to produce expression of microdystrophin

Entrada Therapeutics, which received early funding from CureDuchenne, reported positive data from a Phase 1 ENTR-601-44-101 trial in healthy volunteers. ENTR-601-44, which is designed to skip exon 44, was well-tolerated, and the six individuals in the highest dose cohort (6 mg/kg) demonstrated an average of 0.44% exon skipping after a single dose. This was statistically significant compared to placebo control. Entrada plans to submit regulatory applications in Q4 2024 to initiate separate global Phase 2 clinical trials of ENTR-601-44 for Duchenne amenable to skipping exon 44, and for ENTR-601-45 for Duchenne amenable to skipping exon 45. They also plan to submit regulatory applications in 2025 for a global Phase 2 for Duchenne amenable to skipping exon 50.

Link to press release: https://ir.entradatx.com/news-releases/news-release-details/entrada-therapeutics-reports-positive-preliminary-data-healthy?fbclid=IwZXh0bgNhZW0CMTEAAR1bDCkzTav3cEf10GRT4lBM8O6nrTqgmqjPUijeQAu-8llDFSmxR1E4r_8_aem_fqvvt_8H7EASgVpZjmkrgQ

REGENXBIO announced they are expanding their AFFINITY DUCHENNE Trial, which delivers a micro-dystrophin via AAV8, to include a new cohort of younger patients. The trial, which has been enrolling ambulatory boys with Duchenne aged 4 to 11, is now expected to enroll up to five individuals aged 1-3 as well. These younger participants will receive RGX-202 at the pivotal dose level (dose level 2, at 2x1014 genome copies/kg body weight). REGENXBIO expects to share initial strength and functional assessment data for individuals already treated (at both dose levels) later this year.

Press Release here:
https://www.prnewswire.com/news-releases/regenxbio-announces-expansion-of-affinity-duchenne-trial-to-include-a-new-cohort-of-younger-patients-302180017.html?fbclid=IwZXh0bgNhZW0CMTEAAR2og8RVb65DmhCLRonxCj955EwcjIeFx3nKenw6tO7J8kyIyqB6hzbAB3Q_aem_jGFqPlMGPDyZLQWp1Onrig

Elevidys DMD gene therapy now FDA-approved for ages 4 and older | Label expansion will make one-time treatment available to more patients

Elevidys DMD gene therapy now FDA-approved for age 4 and older The FDA has expanded its approval of Elevidys gene therapy to Duchenne MD patients ages 4 and older regardless of their ability to walk.