October 2016 Newsletter


 
 
Jett Foundation played an important role in the FDA approval of the first therapy for the treatment of Duchenne, Exondys 51, and therefore, so did you. With every dollar you donated and every fundraiser you attended, you helped the Duchenne patient community reach this historic moment in Duchenne history. We thank you, our supporters and allies, for partnering with us on this rugged and emotional journey.
 
Although we delight in this first ever approval for Duchenne, we are continually reminded that only 13% of our community will be eligible to take advantage of Exondys 51. That still leaves many in our patient community without access to a safe and effective treatment. Our zeal to change that continues unabated. Jett Foundation plans to double our efforts to provide empowering education to families, enriching direct service programs and activities for our community, while forging forward with our advocacy efforts to ensure all patients and families affected by Duchenne have access to a safe and effective FDA approved therapy as soon as possible.
 
How can you help?  Get involved - read below about our exciting initiatives and events; save the date for our 1st Annual Jett Foundation Gala , or donate today
so that we may continue to fill the unmet needs of the Duchenne community through advocacy, adaptive activities and education.




Best, 
christine signature
Christine McSherry, Jett's Mom
Executive Director, Jett Foundation


 
THANK YOU!
Gals for Cal 2016 Triathlon
 
On September  11th, 2016 in Hopkinton, MA, more than 70 "GALS" participated in the Max
Performance Title 9 Triathlon. Gals for Cal made history this year with  fundraising total of over $70,000!!! Funds raised will support Jett Foundation's Livin' Large Program, which will help patients and their families take part in activities they otherwise would not be able to. Please consider joining this incredible group  of woman or supporting their effo rts next year.
 
 Click here to learn how you can be part of something "Large"
"Challenging ourselves, challenging Duchenne"
 

#HugsForDuchenne

During the month of September, Jett Foundation teamed up with ICUC Social to launch a social media campaign to raise awareness for Duchenne, #HugsforDuchenne. Duchenne robs kids of the ability to walk, run, feed themselves, and even HUG! 

Why HUGS? This opportunity allowed us to educate the general public that Duchenne affects every muscle in the body, leaving those affected not only unable to walk but also unable to hug. Thanks to all who hugged and helped us raise awareness! Click here to see all the HUGS!



Komedy for a Kause
 
Mark, Troy and Andrew Chaupetta
 
Lots of laughs....huge success! 

Thank you Mark, Troy and Andrew Chaupetta for organizing this successful event raising nearly $30,000!! 

All proceeds form this event benefit Jett Foundation's new program, the HeartShip Fund.  This direct service program, which will be launched in 2017, will help patients and families affected by Duchenne with vital equipment needs.  Save the date for next year - 9/29/17 

Jett Foundation Regional Roundtables
Did you know there are nearly two dozen currently enrolling clinical trials for Duchenne?
Jett Foundation's Regional Clinical Trial Roundtables are an educational program that brings resources and information on treatment options and clinical trials directly to families affected by Duchenne in many cities and towns each year.  Patients, their families and their caregivers are invited to this FREE interactive program where childcare and lunch is provided.  Please click here for additional information and for 2017 dates and locations.
 
Our next Roundtable is scheduled for November 5th in Rochester, NY. 
Save the Date...Upcoming Events 
 
Jett Foundation's First Annual...
 
Shine a Light on Duchenne Gala
The Museum of Science | Cambridge, MA
Thursday, January 26, 2016 
 7:00PM - 11:00PM

For additional information on the event, tickets, or sponsorship opportunities please visit or contact:
 (781) 585-5566

Exondys 51: What's Next?

In the light of the recent approval of Exondys 51, Sarepta Therapeutics has launched a
 new patient support program, SareptAssist, for those seeking information on this newly approved treatment. P atients and families who are affected by Duchenne and may be amendable to Exondys 51 should watch  
If you or someone you know may be amenable to exon skipping or has questions
concerning access to Exondys 51 please  contact Sarepta's case managers:
1 -888-SAREPTA

Opening of New Duchenne Care Center
Stony Brook Children's Hospital in Stony Brook, New York, recently opened a Comprehensive Care Center dedicated to patients with Duchenne muscular dystrophy.  Stony Brook will provide care for the nearly 1,000 patients with Duchenne in the New York, New Jersey, Connecticut regions and beyond. Jett Foundation's fellow Duchenne Alliance member,  Hope for Javier, is responsible for the creation newest center. 

Congratulations Hope for Javier, and thank you on behalf of the entire Duchenne community! 

Click here for additional information regarding the Stony Brook's Duchenne Comprehensive Care Center.

The Latest Industry Partner News
Jett Foundation collaborates with many industry partners who are working towards potential treatments for Duchenne.  Read more about their recent updates below.  For additional information on Duchenne clinical trials go to  Clinicaltrials.gov
 
PTC Therapeutics  provides regulatory update of ataluren for nonsense mutation Duchenne. 

Catabasis Pharmaceuticals completes target enrollment for Part B of the MoveDMD® trial, a phase 2 trial of edasalonexent (CAT-1004) for the potential treatment of Duchenne muscular dystrophy. To learn more read Catabasis monthly newsletter.  

Sarepta Therapeutics and Summit Therapeutics plc announce that they have entered into an exclusive license and collaboration agreement granting Sarepta rights in Europe to Summit's utrophin modulator pipeline for the treatment of Duchenne muscular dystrophy.

Santhera Pharmaceuticals starts phase III study (SIDEROS) with Raxone in patients with Duchenne muscular dystrophy using glucocorticoids.

Catabasis Pharmaceuticals and Sarepta Therapeutics announce a joint research collaboration in Duchenne muscular dystrophy. 

Summit Therapeutics plc. receives fast track designation for ezutromid in the treatment of Duchenne muscular dystrophy. 

Sarepta Therapeutics Announces that the FDA has granted EXONDYS 51™ (eteplirsen) infusion, an exon-skipping therapy to treat Duchenne muscular dystrophy patients amenable to skipping of exon 51, accelerated approval. 

Summit Therapeutics plc launches online resource on utrophin modulation for Duchenne muscular dystrophy community. 

Santhera Pharmaceuticals announces financial results for the first half year 2016. 

Capricor Therapeutics completes enrollment of HOPE-Duchenne clinical trial. 

 
In Loving Memory of Darius Weems
Darius Weems, of Darius Goes West: the Roll of his Life, a well known disability rights activist who brought awareness, funds and smiles to the Duchenne community, passed away on October 9th due to Duchenne related heart failure. Darius was 27 years-old. Darius Goes West, an award winning film available on Netflix that documented Darius' epic, 7,000 mile trek across the country, from Athens Georgia to Los Angeles, inspired thousands of people all over the world. 
 
 Awareness and inspiration weren't Darius' only accomplishments, you can read more about him and his victories here -  http://www.dariusgoeswest.org/

 
   
While we mourn the loss of Darius, an inspiration to so many and a friend to all in the Duchenne community, we ask that  you recall the words he rapped when  his foot touched the Pacific Ocean for the first time: 
 
"I was smiling that day and I'm gonna be smiling when I leave. 'Cause when I die, folks ain't gonna say 'Darius gone'... they gonna say 'Darius gone West'."
 
Darius' loving spirit will never be forgotten, and our thoughts are with the Weems family. 

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