<div>In 2022, Margaret and Bridget were diagnosed with Sanfilippo Syndrome, a neurodegenerative disease that is like Alzheimer's in children. They will slowly lose all the skills they once had.
</div>
<div>Currently, there is no FDA-approved treatment or cure for Sanfilippo Syndrome.
</div>
<div>But there is hope.
</div>
<div>It’s real-and it could change everything for children with Sanfilippo Syndrome 3B. But we need to move fast.
</div>
<div>The Sanfilippo patient community has been offered a unique opportunity by a drug manufacturing company to receive enzyme replacement therapy through an Expanded Access pathway. This is not the typical way for Expanded Access to be rolled out; however, given the extreme unmet need, we are grateful for any prospect to access treatment. This opportunity requires the patient families along with the related Sanfilippo foundations to secure the funding.
</div>
<div><br />
</div>
<div>There are many details that remain unknown, including the criteria for children to be selected for the Expanded Access, the site locations, the exact timing, and much more. Eligibility and enrollment will be determined by doctors and researchers working with the pharmaceutical company.
</div>
<div><br />
</div>
<div>Potentially, this will supply up to 14 children with one year of enzyme treatment. The path beyond that is uncertain. The hope is that the therapy will become FDA approved during this time and patients would then be able to be covered by insurance to receive the approved drug. However, the path to FDA approval can be a challenge with delays being very common, and there is no guarantee of approval.
</div>
<div><br />
</div>
<div>When thousands give a little, miracle’s happen. Every share, every dollar, every prayer matters!
</div>
<div><br />
</div>
<div><br />
</div>
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</div>

Gerard Cunningham

Rob King

JOHN-MIKE MCAULIFFE

Anonymous

Gerald Crossan

Anthony Rotondo

Vincent Murphy

Connor Catapano

Michael Greenfield

Bill Carlson

Charlie Larocchia

Nicholas D'Ambra

Elise Otton

Kayla Schmidt

Travis Wahl

Cure Margaret & Bridget Lindquist

platform

Platform is iOS

Android

Adyen Google Pay Development Audience

$opt_dummy_attribute

English only

Deprecate Stripe Legacy

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Adyen V6 Bene Transfer & MyMoney Campaign Organizers - test01

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fund country is US and user language is English

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desktop

Android v12.11.0 and above

Platform is Android

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Payment method type group to be vaulted on success

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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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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

Mexico country code

Posters INTL

NPO Profile Opted-In

iOS at or above 12.4.0

non-US recurring donors

Canada Campaign Owner Group

Fundraiser Without Meta Link

Scheduler payment tester gorup

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Charities allow to be claimed unclaimed (PRD)

Canada (CA) users

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UC Tipping v20250115 International

pymk_production_xplr_fe_team

Android at or above 12.2.0

Profile photo entry point audience

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Activity Tracking v1 Beta Testing

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start gfm intl allowed locales

Platform is iOS v11.2.0 and above

unclaimed charities

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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

UC Tipping v20250107

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Influencer - Prod

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Auto Thanks V3 Rollout Audience

Thank you V3 testing whitelist

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Logged In Users

CH Donation Upsell audience

Android v10.6.0 and above

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v11.5.0 and above

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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

Cure Margaret & Bridget Lindquist

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