July 2016 Update


July is off with a bang - Happy Independence Day to all. To those of us living with Duchenne, independence has another meaning and the Jett Foundation is fighting everyday for all of those who are losing their ability to run, to walk, to hug.

The magic of Camp Promise 2016 is underway! Our Camp Director has been planning all year for these 3 amazing weeks - and we can measure the success in all these  happy camper smiles.

While we still eagerly await FDA's final decision on eteplirsen, Jett Foundation will continue to work to expedite the development and approval of candidate therapies until every child born with Duchenne has access to a safe and effective treatment. 

How can you help?  Donate today so that we may continue to fill the unmet needs of the Duchenne community through advocacy, adaptive activities and education.

Thank you to everyone for your support. 

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

Resignation Announcement
It's with regret that we announce the resignation of Jenn McNary as Program and Outreach Director at Jett Foundation. Jenn has been a fierce advocate and an integral member of the Jett Foundation team over the past 3 years and we are grateful for all her contributions. It is with the warmest of personal regards that we wish Jenn success as she begins a new and exciting chapter in her career.
Camp Promise East
Jett Foundation's Camp Promise East made 21 VERY happy Campers this week!  Camp Promise is "the best week of the year" says many of our campers.  Camp is a welcoming community where campers learn from and support each other, fostering new and lasting friendships that extend beyond camp. We are committed to providing Camp Promise in a professional, safe, and respectful environment with a leadership team, trained volunteers, and licensed medical staff who support the needs of each and every camper.

Check out photos from Camp Promise East
Jett Adaptive Sailing Camp

Jett Foundation Regional Roundtables

Our Regional Roundtable program continues to be a great resource to families and caregivers.

Our next Roundtable is August 27th in Lafayette, Louisiana. 

Click here for more info
Get Involved!  Join a Jett Team!
jettride 2010 logo July22-30th
Teens (14-18)from across America will make a three state (MA,NY,VT) tour this summer to help raise funds and awareness for Duchenne.  Do you know a teen looking for a life changing/saving experience? 
July 31, 2016
Registration is now open - join our team
of men and women "TRI-ing" to end Duchenne.

Gals for Cal,
Title 9 Triathlon
Sept. 11th Hopkinton, MA

Join this amazing team of women who are swimming, biking and running for Duchenne.  Join our team today! Visit our website for more info.

Industry Partner News
The Jett Foundation is thrilled to collaborate with so 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

Santhera Pharmaceuticals  has announced that it has received written correspondence from the U.S. Food and Drug Administration (FDA) on its proposed subpart H approval pathway for Raxone in DMD patients not taking concomitant glucocorticoids. Santhera had proposed that the planned SIDEROS trial would provide confirmatory evidence of efficacy in these patients whilst expanding the label to include the treatment of glucocorticoid-using patients.  From its review of this strategy, the FDA concluded that results from the SIDEROS trial, which is powered to detect a difference in the established surrogate endpoint Forced Vital Capacity percent predicted (FVC%p) in glucocorticoid-using patients, should be provided at the time of filing to support an NDA for the treatment of DMD patients irrespective of their glucocorticoid use status.

Catabasis Pharmaceuticals announces the Initiation of an Open-Label Extension for the MoveDMD® Trial Studying Edasalonexent (CAT-1004) in Duchenne Muscular Dystrophy. If you have any questions regarding Catabasis Duchenne programs to go https:// clinicaltrials.gov/ct2/show/NCT02439216?term=catabasis&rank=3   
PTC Therapeutics initatiates Phase 2 Clinical Trial of Translarna™ (ataluren) for Treatment of Nonsense Mutation Duchenne Muscular Dystrophy in Pediatric Patients Between the Ages of Two and Five Years.  If you have any questions for PTC go to:  https://clinicaltrials.gov/ct2/show/NCT02369731?term=PTC+duchenne&rank=12

Capricor Therapeutics announced that patient enrollment in its ongoing randomized HOPE-Duchenne clinical trial (Halt cardiomyOPathy progrEssion in Duchenne) has exceeded 50% of its 24-patient target. Capricor expects the trial to complete its enrollment in the third quarter of 2016, and to report top line data from HOPE-Duchenne in the first quarter of 2017.  If you have questions about Capricor's Duchenne programs go to: https://clinicaltrials.gov/ct2/results?term=Capricor&Search=Search

Sarepta Therapeutics  is updating the Duchenne community regarding recent changes to the Phase III confirmatory clinical study, ESSENCE (Study 4045-301), as posted on clinicaltrials.gov. [Identifier: NCT02500381].  ESSENCE will serve as a confirmatory study for eteplirsen if it is granted accelerated approval. Post-marketing confirmatory studies are a requirement for drugs that are granted accelerated approval in order to verify the clinical benefit of the drug.  If you have any questions about Sarepta's Duchenne programs go to https://clinicaltrials.gov/ct2/results?term=sarepta+duchenne&Search=Search  
BioMarin Phamaceuticals update for the Duchenne Community .   BioMarin is providing a further update to the Duchenne Community in light of recent announcements of withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on products BMN 044, BMN 045 and BMN 053.   If you have any questions for BioMarin please contact BioMarin's patient advocacy team (patientadvocacy@bmrn.com).

Summit Therapeutics plc  
announces that it has enrolled the first patient in PhaseOut DMD, a Phase 2 proof of concept clinical trial of ezutromid (formerly SMT C1100) in patients with DMD. Ezutromid dosing is expected to follow a screening period of up to 28 days. If you have any questions about Summit Duchenne programs go to:   https://clinicaltrials.gov/ct2/show/NCT02213263?term=Summit+duchenne&rank=3 
Marathon Pharmaceuticals, LLC, announced it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the investigational drug deflazacort for the treatment of patients with Duchenne muscular dystrophy (DMD), the most common and most severe form of muscular dystrophy. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing. For additional information on Marathon's Duchenne programs go to:    https://clinicaltrials.gov/ct2/results?term=marathon+duchenne&Search=Search 
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