One Donation, Double the Good! We have been working hard the past year, ever since Kevin, Angela, myself and my husband<a href="https://thriving.childrenshospital.org/families-of-girls-with-spg47-find-support-in-each-other/" target="_blank"> first met</a> at Boston Children's hospital. We've had several more equally adorable and sweet youngsters recieve a diagnosis of SPG47: Ethan (3yo), Wren (4yo), Sofia (6yo) and Susu (3yo). Their sweet faces are in the photo below and there stories can be found <a href="http://curespg47.org/our-story.php" target="_blank">here</a> . Their parents are equally committed to finding a cure and have joined the fight. We are lucky to have them. <img class="not-resizable" src="https://d2g8igdw686xgo.cloudfront.net/25559388_1513448272.2534_funddescription.png" alt="" width="100%" /> Every single donation made in the last year has contributed towards launching two promising research options towards stopping or treating the destructive course of <a href="http://www.childrenshospital.org/conditions-and-treatments/conditions/spastic-paraplegia-47" target="_blank">SPG47</a> . From now until January 31st, 2018, total donations up to $100,000 are being matched by an anonymous Angel Donor! Your generous donation will be worth double! Triple if your company matches- be sure to ask. WHY SO URGENT? Degeneration typically begins between 3 to 5-years-old. Paraplegia or quadriplegia, and loss of speech gained, are expected by early teens. With successful treatment, these children would continue to get stronger and work towards independence. Without, they will gradually lose what they’ve worked so hard to attain. Robbie is 3-years-old and distressing signs that the disease is progressing are already presenting on her. <img class="not-resizable" src="https://d2g8igdw686xgo.cloudfront.net/25559388_1511743183.1734_funddescription.jpg" alt="" width="100%" /> WHERE THE MONEY IS GOING <a href="https://www.sheffield.ac.uk/neuroscience/staff/azzouz" target="_blank">Mimoun Azzouz</a> , PhD, Director of Research and Innovation at the University of Sheffield, England, is the principal investigator for our <a href="http://www.danafarberbostonchildrens.org/innovative-approaches/gene-therapy/what-is-gene-therapy.aspx" target="_blank">gene therapy research. </a> <a href="https://connects.catalyst.harvard.edu/Profiles/display/Person/125033" target="_blank">Darius Ebrahimi-Fakhari, MD</a> , in the Sahin lab at Boston Children’s Hospital is leading research using <a href="http://stemcell.childrenshospital.org/about-stem-cells/pluripotent-stem-cells-101/pluripotent-stem-cell-research/research-on-induced-pluripotent-stem-cells/" target="_blank">induced pluripotent stem cells (ipsc).</a> This ipsc study involves testing existing FDA approved drug compounds to see if they can positively influence the effects of the mutated gene responsible for this disease. <img class="not-resizable" src="https://d2g8igdw686xgo.cloudfront.net/25559388_1511743261.2111_funddescription.jpg" alt="" width="100%" /> <img class="not-resizable" src="https://d2g8igdw686xgo.cloudfront.net/25559388_1511743794.84_funddescription.jpg" alt="" width="100%" /> All donations are tax deductible and go to the Cure SPG47, a non-profit 501c3 (Tax ID: 81-4300824) who will make sure that 100% of the funds go to the research, safety trials, and regulatory affairs needed to get this treatment to children and young adults now with this diagnosis, as well as those who have yet-to-be diagnosed. Other ways to donate: Visit <a href="http://www.curespg47.org/donations.php" target="_blank"><a href="http://www.curespg47.org/donations.php" target="_blank" class="" rel="nofollow noopener noreferrer">http://www.curespg47.org/donations.php</a></a> Or send checks payable to Cure SPG47 to: 24R Pleasant St, Unit 2 Newburyport, MA 01950 POSTIVE RESEARCH COULD IMPACT MANY OTHERS We've now met several other children recently diagnosed with SPG47 and believe it is underdiagnosed. Initial assessment of Robbie’s first MRI led to a diagnosis of PVL (<a href="https://outlook.live.com/owa/Periventricular%20leukomalacia" target="_blank">Periventricular leukomalacia</a> ), brain damage often occurring prenatally or at birth. It took our neurologist’s shrewd eye to determine that the cause was likely genetic, spurring us to pursue genetic testing. With a clinical presentation in pediatrics patients remarkably similar to Cerebral Palsy’s <a href="https://rarediseases.info.nih.gov/diseases/9637/spastic-diplegia-cerebral-palsy" target="_blank">spastic diplegia</a> form, there could be many more children with a misdiagnosis. Awareness is key! HOW YOU CAN HELP We need your help to raise research funds. If we raise $100,000, it will be DOUBLED by our Angel donor. This can make a significant impact in our preliminary proof-of-concept research, where any positive outcomes may lead to outside grant funding towards human clinical trials. We are calling on the generosity of family, friends, neighbors and strangers to help us #DoubletheGoodSPG47 With your help, we can reach this goal together and give these children a future that is as bright as their smile and as full as their laughter.   With so much gratefulness, Kasey and Chris Edwards, Kevin and Angela Duffy, and all of the parents fighting beside us HOW YOU CAN HELP TODAY 1. Donate what you can; no amount too small and donations are tax deductible 2. Share on all social media with hashtag #DoubletheGoodSPG47 3. Email our GoFundMe and these steps to all contacts 4. Leverage any avenue you have to make this go viral (or send us leads/tips) Learn more about SPG47 at <a href="http://www.curespg47.org" target="_blank"><a href="http://www.curespg47.org" target="_blank" class="" rel="nofollow noopener noreferrer">http://www.curespg47.org</a></a>  Follow Updates at <a href="https://www.facebook.com/Curespg47/" target="_blank"><a href="https://www.facebook.com/Curespg47/" target="_blank" class="" rel="nofollow noopener noreferrer">https://www.facebook.com/Curespg47/</a></a>    Learn more about the families at <a href="http://www.curespg47.org/our-story.php" target="_blank"><a href="http://www.curespg47.org/our-story.php" target="_blank" class="" rel="nofollow noopener noreferrer">http://www.curespg47.org/our-story.php</a></a>  In the Press <a href="http://people.com/human-interest/toddler-best-friends-same-deadly-disease-parents-search-cure/" target="_blank">PEOPLE Magazine </a> <a href="http://www.bostonglobe.com/metro/regionals/north/2017/11/10/for-players-and-ill-child-making-something-out-nothing/L9LFrdDjpG2lq7FPRowKHM/story.html" target="_blank">The Boston Globe</a> <a href="http://philadelphia.cbslocal.com/2017/09/12/hundreds-turn-out-to-raise-funds/" target="_blank">CBS Philly</a> <a href="http://pulse.seattlechildrens.org/the-comfort-of-a-diagnosis-and-knowing-you-are-not-alone/" target="_blank">Seattle Children's Hospital Blog</a> <a href="http://www.insideedition.com/headlines/20960-2-kids-with-rare-disease-who-live-300-miles-apart-finally-meet-for-special-playdate" target="_blank">Inside Edition</a> <a href="http://www.wcvb.com/article/newburyport-family-fighting-daughter-s-rare-disease/8494692" target="_blank">WCVB Ch. 5 News</a> <a href="http://6abc.com/health/2-toddlers-with-rare-disease-form-friendship-in-their-fight-for-cure-/1660738/" target="_blank">WPVI-TV Philadelphia</a> <a href="https://thriving.childrenshospital.org/families-of-girls-with-spg47-find-support-in-each-other/" target="_blank">Boston Children’s Hospital THRIVING Blog </a> <a href="https://vector.childrenshospital.org/2017/04/spg47-citizen-science/" target="_blank">Boston Children’s Hospital Science and Clinical Innovation Blog</a> <a href="https://www.nbcboston.com/news/local/Special-Books-by-Special-Kids-422662854.html" target="_blank"> NBC Boston </a> A special thanks to The Jackson Lab in Bar Harbor, ME for donating their time, effort and money in funding a knock-out mouse model that will be essential in progress with gene therapy research.

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Anonymous

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Holly Dinges

Diane  Betz

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Debbie Stanish

The Perruccis

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Paula and Joe  Metivier

Roger and Kwi Murray

Eric Humphrey 

Amy  Brackenbury 

Susan Bernard

One Donation, Double the Good

platform

Platform is iOS

Android

Adyen Google Pay Development Audience

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Deprecate Stripe Legacy

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Adyen V6 Bene Transfer & MyMoney Campaign Organizers - test01

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Platform is Android

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EUR Donation Upsell

Share Text generation Skip Fundraiser

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Payment Identity Transfers ATO Risk Check (Targeted) - Production

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Version 9.2.1 and above

AUD Donation Upsell audience

fundraiserCountryCode is US/EN

Active Fundraiser - Smart Default Cadence

DAF Internal

Vault on success card payment method

Platform is iOS v11.0.0 and above

Desktop users

Person ids

United States (US) users

allow yuno fundraisers for test

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non-US recurring donors

Canada Campaign Owner Group

Fundraiser Without Meta Link

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Charities allow to be claimed unclaimed (PRD)

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UC Tipping v20250115 International

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Android at or above 12.2.0

Profile photo entry point audience

smart goals (fundraiser/manage) on test01

Activity Tracking v1 Beta Testing

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start gfm intl allowed locales

Platform is iOS v11.2.0 and above

prod_ach_audience

AI Chatbot Audience

amp13 only new funds (prod) (anyword auto)

UC Flow Enabled (Prod)

Payment Identity Beneficiary Verification - Production

shared notification bell audience

Custom users

ach frontend audience

Android v11.0.0 and above

Users Receiving Payments Paused Notifications

smart goals test01

Manage Coach - US English Organizers Only + new funds only

Team EGG - IP Address list

Fundraiser Without Instagram Deep Link

Native 11.4.0 and above

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Influencer - Prod

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Native 11.4.0 and higher

New funds eligible - Video Sharing (PROD)

GB Donation Upsell audience 

Auto Thanks V3 Rollout Audience

Thank you V3 testing whitelist

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CH Donation Upsell audience

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Survicate

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iOS App Version

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PYMK has donors

iOS v12.3.0 and above

One Donation, Double the Good

Still $89,350 to go. Help us build momentum.