1. CureDuchenne Organization Wikipedia Page

2. Overview

3. Duchenne Muscular Dystrophy

4. History

5. Organization
     Impact Since 2003:    Mission:    Founder's Message:    CureDuchenne Ventures:    CureDuchenne Cares:    DuchenneXchange:  

6. Scientific Partnerships
     Treating the Whole Disease    Drug & Medicine Research    CureDuchenne-funded research projects:    Current research:    Events    Napa Wine Series    Futures    Getzlaf Golf Shootout    Champions to CureDuchenne    Blingo    Dealing for Duchenne    Love Cures    [https://musculardystrophynews.com/2018/10/31/cure-duchenne-opens-inaugural-pinkies-up-to-cureduchenne-fundraising-campaign/ Pinkies Up to Cure Duchenne]    Partnerships (celebrities, etc.)    Clay Matthews    Ryan Getzlaf  

7. References

8. CureDuchenne Organization Wikipedia Page

9. CureDuchenne Organization Wikipedia Page Resubmission

CureDuchenne Organization Wikipedia Page

Overview

Founded with the goal of finding a cure for Duchenne muscular dystrophy, CureDuchenne utilizes a venture philanthropy model to fund new research, early diagnosis and treatment access. Additionally, CureDuchenne hosts nation-wide education and support programs as well as resources to support Duchenne patients, their families, caregivers and healthcare providers.

Founded by Paul and Debra Miller in 2003, after their son was diagnosed with the disease, CureDuchenne combines fundraising and venture philanthropy, leveraging donor dollars to maximize support for promising research into effective treatments for those suffering from Duchenne. Investment proceeds are redeployed to support research and other mission-critical programs.

Duchenne Muscular Dystrophy

The most common and fatal form of muscular dystrophy, Duchenne is a devastating, progressive muscle-wasting disease most commonly found in young boys. Affecting 15,000 children in the US and 300,000 worldwide, there is currently no known cure for Duchenne.

Muscular dystrophies are a group of genetic disorders characterized by muscle weakness. Duchenne is the most common and severe form, caused by loss of dystrophin, beneath the sarcolemma. The molecular mechanisms of the disease have been extensively investigated since the discovery of the gene in 1986.

• Caused by a loss of dystrophin, a protein that helps keep muscles intact.
• Early stages make simple tasks difficult.
• Later stages cause failure in heart and breathing muscles.
• Most children suffering from Duchenne are wheelchair-bound by age 12.
• Many don’t survive beyond their 20s.

History

In 2003, Paul and Debra Miller received the diagnosis that their 5-year-old son Hawken had Duchenne muscular dystrophy. At the time, there was no treatment for Duchenne — the only course of action was to prepare for the progression of the disease. They were told Hawken would likely not live beyond his early twenties.

Despite this prognosis, the Millers began raising funds to invest in scientific research to help accelerate drug development for Duchenne. From this, CureDuchenne was born.

In the years since, CureDuchenne has partnered with research firms and institutions around the world to develop treatments and avenues towards a cure.

Organization

Impact Since 2003:

• Over $30 million raised for research and development of impactful programs that help Duchenne families.
• 12 sponsored projects that have progressed to human clinical trials.
• Provided early funding for research into the first FDA-approved Duchenne drug — Eteplirsen, developed by [https://www.sarepta.com/ Sarepta].
• Between 81% and 91% of every dollar raised is used to fund research and direct mission-specific programs to save lives.

Mission:

The mission of CureDuchenne is to find a cure for Duchenne muscular dystrophy - "CureDuchenne is recognized as the global leader in research, patient care and innovation for improving and extending the lives of those with Duchenne".^[1]

To enable progress for drug development and extend ambulation for patients, CureDuchenne collaborates with pharmaceutical companies, medical and healthcare professionals, their in-house scientific advisory board, and board of directors.

Founder's Message:

“Like other parents with a child with Duchenne, we’ve been riding an emotional roller coaster since our son was diagnosed. The joys of our son’s smile and determination take us to the top of the world, and then Duchenne muscular dystrophy hits again, and again.

Each parent experiences this emotional roller coaster, and we are bound together by the journey as we search for better care and, ultimately, a cure.

CureDuchenne was formed in response to the precise needs of those who live with Duchenne and their families. From creating the first Duchenne-specific physical therapy program to funding enabling studies for human clinical trials for eteplirsen, the first-ever FDA approved drug for Duchenne patients, we believe in fighting Duchenne from every angle. At CureDuchenne, we care about you, your children and the quality of the life you live together. And we are striving to make the future better.

It’s been our philosophy from the beginning to pursue better medical and therapeutic care, band together to find a cure and build a stronger Duchenne community. Saving children affected by Duchenne has become our life’s work, and together we can be the best advocates for our children.”

CureDuchenne Ventures:

CureDuchenne Ventures supports Duchenne research across the globe by leveraging venture philanthropy to encourage new drug and treatment development. The proceeds from investments are deployed towards next-gen research to maximize impact of donations. CureDuchenne Ventures identifies, funds and fosters promising early-stage science to find a cure for Duchenne. 100% of proceeds from investments are redeployed to support research into next-generation technologies.

CureDuchenne Cares:

CureDuchenne Cares is an interactive education and outreach program where patients, families and healthcare professionals can get information, resources and training to improve patients’ lives. Hosting over 20 workshops and conferences nationally, CureDuchenne Cares is free for families and caregivers.

• 26 CureDuchenne Cares educational events
• 370 physical therapists trained
• 1000+ parents, patients and caregivers attended CureDuchenne Cares educational events since the program’s inception
• 1,100 estimated number of patients receiving care from a CureDuchenne-trained physical therapist (Average 3 patients per physical therapist).
• 16 biotech and pharmaceutical company partners in 2018
• 26 CureDuchenne Cares-[https://www.cureduchenne.org/care/find-certified-therapist/ certified physical therapists]

DuchenneXchange:

“Built by the Duchenne community for the Duchenne community,” [https://www.duchennexchange.org/ DuchenneXchange] is a free, collaborative online social platform that gives patients, families, caregivers and advocacy organizations a place to connect and share knowledge. DuchenneXchange provides the entire Duchenne community with the most current education, peer support, curated resources, clinical trial information and news.

Scientific Partnerships

CureDuchenne provided funding for the 1st FDA-approved drug for the treatment of Duchenne: Eteplirsen, or EXONDYS 51. 13% of Duchenne patients are amenable to exon skipping 51, meaning they can utilize this treatment.

“It was a long and complicated road, however, to this moment…A key issue was whether the drug can sufficiently produce higher levels of a protein called dystrophin. Without this protein, muscle fibers degenerate and voluntary movement becomes impossible.” -Ed Silverman, STAT^[2]

CureDuchenne also provided early funding to [https://www.sarepta.com/ Sarepta] to help move this research into human clinical trials.

Treating the Whole Disease

While there may be no single cure for Duchenne muscular dystrophy, current science indicates that by working towards treatments and cures for the disparate effects of Duchenne, effective solutions can be uncovered to improve the lives of those afflicted by this disease.  

Effective area of focus:

• Exon skipping and Stop Codon Read-Through
• Gene Editing
• Gene Therapy
• Protein Replacement
• Cardiac
• Anti-inflammatory
• Anti-Fibrosis
• Advocacy
• Physical Therapy and Education
• Clinical Trials
• Patient Data Analysis

In October, 2018, CureDuchenne partnered with Baebies — a company that develops innovative products and services for newborn screening and pediatric testing.

“As part of the deal, Baebies will add Duchenne muscular dystrophy, the most common and lethal form of muscular dystrophy that primarily affects young boys, to its expanded newborn screening service. It’s estimated that one in 5000 male newborns are born with the disease. Newborn screening allows for early diagnosis, which is beneficial for patients and their families.” -Chantal Allam, WRAL Tech Wire^[3]

Dr. Eric Olson and his team at Exonics, a CRISPR/Cas gene editing company CureDuchenne helped form, published data that showed a genetic correction of the dystrophin gene led to an increase in dystrophin by 92% in the heart of canines with Duchenne.

“In a study published Thursday in Science,^[4] a team led by Eric Olson at the University of Texas Southwestern Medical Center used Crispr to successfully modify the DNA of four young dogs, reversing the molecular defect responsible for their muscle wasting disease.”^{[5] [6]}– Megan Molteni, Wired

“CureDuchenne has helped raise funding for research into the use of CRISPR-Cas9 to find the mutated gene and correct it. A paper on the use of this gene therapy in animals, published last October, found that this particular use of CRISPR-Cas9 restored dystrophin levels in the heart of one dog to 92 percent of normal levels^[7].”^[8] – Anna Almendrala, Huffington Post

Exonics has licensed the gene editing technology developed by Olson’s lab and is moving it toward human trials, with the hopes of one day commercializing treatments. The partnership — which focuses on developing gene editing technologies, like CRISPR/Cas9^[9] — began early in 2017.

“If the treatment can stop muscular dystrophy in dogs, that would set the stage for giving the experimental treatment to boys, according to the research team.”^[10] – Antonio Regalado, Technology Review

Drug & Medicine Research

CureDuchenne-funded research projects:

• [https://www.pfizer.com/news/press-release/press-release-detail/pfizer_doses_first_patient_using_investigational_mini_dystrophin_gene_therapy_for_the_treatment_of_duchenne_muscular_dystrophy Bamboo (gene therapy)]
• [https://www.marketwatch.com/press-release/biomarin-transfers-duchenne-natural-history-database-to-cureduchenne-to-improve-knowledge-of-duchenne-patients-and-improve-clinical-outcomes-2017-01-10 Biomarin (exon skipping)]
• [https://globalgenes.org/raredaily/cure-duchenne-ventures-llc-provide-1-million-capricor-therapeutics-advance-promising-research-treat-heart-disease-associated-duchenne-muscular-dystrophy/ Capricor Therapeutics (cardiac stem cell derived therapy)]
• [https://www.businesswire.com/news/home/20181001005209/en/cTAP-Present-Late-breaking-Results-Supporting-Advancements-Clinical Collaborative Trajectory Analysis Project (statistical analysis of natural history studies)]
• [https://www.genengnews.com/topics/genome-editing/cureduchenne-ventures-commits-5m-to-startup-with-crispr-based-dmd-therapy/ Exonics Therapeutics (gene editing/CRISPR)]
• MyoTherix, Inc. (anti-inflammatory, anti-osteoporosis, potential steroid-sparing therapies)
• Nationwide Children's (exon skipping [duplication exon 2 mutations])
• PTC Therapeutics (stop codon read-through)
• RASRx (anti-fibrotic)
• Sarepta (exon skipping)
• Summit (utrophin upregulation)
• Trinds (neuromuscular-focused contract research organization)

Current research:

Events

Every year, CureDuchenne sponsors events and initiatives not only to raise funds for research and courses, but also to help promote awareness of Duchenne muscular dystrophy.

Napa Wine Series

With events in Newport, CA, and Boston, MA, CureDuchenne congregates premier Napa Valley vintners and high-end chefs for an evening of wine tasting, dining and auctions. Proceeds go towards raising valuable funds for CureDuchenne programs. Past events have been held at the [https://www.monarchbeachresort.com/ Monarch Beach Resort] in Napa and The Langham in Boston.

Futures

In an effort to provide more immersive educational experiences for the Duchenne community, [https://musculardystrophynews.com/2018/10/10/cureduchenne-plans-futures-event-nov-3-4-boston/ CureDuchenne hosts CureDuchenne Futures], a free conference where families, patients and caregivers can learn about best care practices and hold face-to-face meetings with leading scientists, clinicians and experts.

“Caregivers of Duchenne muscular dystrophy and patients suffering from the rare disease on the east coast will have an opportunity to learn about the best practices of care for Duchenne at a conference titled ‘Futures’ hosted by nonprofit CureDuchenne. ‘We are very excited to deliver Futures to the Duchenne community,’ said Debra Miller, founder and CEO, CureDuchenne. ‘With a focus on positive futures for all in the Duchenne community, this conference is designed to provide Duchenne families with the information and motivation they need to live the best and longest life possible.^[11]’”

“‘Futures is going to meet the needs of the community,’ [Dr. Brenda] Wong [MD, University of Massachusetts Medical Center] said in a press release emailed to [https://musculardystrophynews.com/2018/10/10/cureduchenne-plans-futures-event-nov-3-4-boston/ Muscular Dystrophy News]. ‘Not only will it cover the latest news in gene therapy and editing, clinical trials and steroid use, but will also offer insightful topics that are important to staying mentally healthy and focused on living your best life for Duchenne patients and caregivers.’”

Getzlaf Golf Shootout

Hosted by Ryan Getzlaf, captain of the Anaheim Ducks, this two-day charity golf event. “brings together athletes, celebrities and community leaders in support of CureDuchenne^[12].”

Champions to CureDuchenne

An annual event set in Austin, TX, Champions is an evening for the local community to gather for food and activities, and to raise funds for CureDuchenne. To date, this event has raised $1.7 million.

Blingo

Hosted in Philadelphia, Blingo brings together the Duchenne community and local celebrities for an evening of gaming to raise funds for Duchenne research.

“Blingo, an annual event hosted by the Gambhir family and co-hosted with Jesse and Beka Rendell and Governor Ed Rendell took place at Loews Hotel in Philadelphia on Saturday, October 13, 2018. It was a very well attended night to benefit a very serious mission – CureDuchenne. Former Eagles player Tra Thomas was the MC and the honorary chair was Ed Rendell.”^[13]

“A live pledge raised over $60,000 for the cause and was followed by a live auction.”^[14]

Dealing for Duchenne

CureDuchenne’s San Antonio fundraising event, Dealing for Duchenne, is an evening of gaming with the goal of raising funds for CureDuchenne programs.

Love Cures

A San Diego-based event, Love Cures brings together the Duchenne community, caretakers, educators, families and friends for an enjoyable evening of food, music and activities to raise money for vital research.

[https://musculardystrophynews.com/2018/10/31/cure-duchenne-opens-inaugural-pinkies-up-to-cureduchenne-fundraising-campaign/ Pinkies Up to Cure Duchenne]

A social campaign wherein people paint their pinky nail blue to raise awareness of and funds for Duchenne muscular dystrophy. To participate, people simply paint their pinky nail blue and make a donation to CureDuchenne.

“A campaign hopes to raise awareness of Duchenne muscular dystrophy (DMD) and promote research toward a cure by asking supporters to paint a nail on one of their little fingers blue. Called '[https://www.cureduchenne.org/pinkiesup/ Pinkies Up to CureDuchenne],' the inaugural effort is led by [https://www.cureduchenne.org/ CureDuchenne]..."^[15]

Partnerships (celebrities, etc.)

Clay Matthews

Since 2011, NFL linebacker Clay Matthews has worked with CureDuchenne to raise awareness and funds for the cause. From appearing in PSAs and at fundraising events to bringing Duchenne kids to Lambeau Field, Matthews has become one of the largest personas in the Duchenne world.

“As a professional athlete, I can’t imagine having to deal with many of the things that these boys deal with on a daily basis. I work hard to build my muscles, but these boys don’t get that choice. Instead, boys with Duchenne, and their families, can only watch as their muscles get weaker on a daily basis.I was compelled to help because these boys and this cause deserve a voice. Moreover, they deserve a cure.”^[16]

“Clay Matthews, the Super Bowl-winning linebacker for the Green Bay Packers, became a part of the rare disease community almost by chance. His first exposure to Duchenne, a rare muscle degeneration disease that affects boys, was for a CureDuchenne PSA that he lent his likeness to. That year happened to be the year Matthews and his team won the Super Bowl.The win meant the PSA received plenty of airtime, and Duchenne gained significant awareness. In the months that followed, Matthews found himself drawn into a tight-knit community that was searching for a cure.”^[17]

Additionally, he has started his own initiatives, including the “[https://www.ispot.tv/ad/wC43/nfl-my-cause-my-cleats-combat-duchenne-featuring-clay-matthews My Cleats, My Cause]” campaign, where he wore custom-made cleats designed by boys with Duchenne.

“Clay Matthews of the Green Bay Packers took to the field in Week 13 of the NFL season wearing a cause close to his heart on his feet, as he joined the football league’s “[https://www.packers.com/video/matthews-1-on-1-my-cause-my-cleats-18193832 My Cause My Cleats]” campaign to raise funds for [https://musculardystrophynews.com/tag/duchenne-muscular-dystrophy/ Duchenne muscular dystrophy (DMD)]…^[18]Matthews also [https://secure3.convio.net/cd/site/Donation2;jsessionid=00000000.app357a?3961.donation=form1&df_id=3961&mfc_pref=T&NONCE_TOKEN=8D251A237629BA4A0FC327A361D8E1FB matched donations] of up to $25,000 received by his campaign between Dec. 2 and 4, with money going to fund research into DMD and programs helping members of the DMD community. According to a press release, Matthews is a supporter of CureDuchenne since 2010, when he recorded a public service announcement (PSA) for the organization. He has since then corresponded with boys with DMD, aiming to give them hope for their future.”^[19]

Ryan Getzlaf

Since 2011, Anaheim Ducks’ captain Ryan Getzlaf and his wife Paige have hosted the annual The Getzlaf Golf Shootout and MVP Party. This fundraising event brings together athletes, celebrities and community leaders in support of CureDuchenne.

"‘Each year as we take to the course for our annual shootout, I am humbled by the amount of support we receive from a variety of people and organizations,’ said Ryan Getzlaf. ‘It is important that we continue to fight for these children's lives and every registration, donation and sponsorship makes a difference in saving those affected by Duchenne. Together, we will continue to fight for a cure.’"

References

CureDuchenne Organization Wikipedia Page

CureDuchenne Organization Wikipedia Page Resubmission

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