Rare As One Network Cycle 1 Impact Report

Rare As One Network Cycle 1 Impact Report

@HeidiBP

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1 week ago 390

Science #RareAsOne #Patients #Research

Rare As One Network Cycle 1 Impact Report

@HeidiBP1 week ago

1
DECEMBER 2024
RARE AS ONE
NETWORK CYCLE 1
IMPACT REPORT

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Introduction 2
01
Introduction
4 Welcome
10 Impact At A Glance
11 Publications
12 CZI Support…

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Introduction 3
Ann Geffen (left), Executive Director of the TANGO2 Research Foundation, and Medha-…

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Introduction 4
Welcome
Dear reader,
Today, approximately 10,000 rare
diseases collectively aff…

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Introduction 5
(ii) convening their patient and research
communities; (iii) aligning research
…

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6
One Network as an incubator-style
program to help build the capacities
of the organizations …

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Introduction 7
Julie Raskin (left) CEO, and Mahlet Mesfin (right), former Research Manager,of Cong…

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Introduction 8
animal models that are enabling critical,
data-driven insights into rare disease …

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Introduction 9
Left to right: Andra Stratton, Heidi BjornsonPennell, and Tania Simoncelli.
Steph…

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Introduction 10
Impact At A Glance
24
Organizations developed or
contributed to the developmen…

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Introduction 11
Publications
During their grant period, the
organizations of the Rare As One
…

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Introduction 12
CZI Support and
the Network Effect
RARE AS ONE
NETWORK
In addition to grant …

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Introduction 13
“ “
“As a RAO Science Panelist, I found the interaction
between physician-scien…

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Introduction
“ “ “ “
14
“In a few short years, newly formed
entities have established multi-st…

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RAO Network Cycle 1 15
Case Studies
Science in Society grantees, including members of the Rare As…

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16
APBD Research Foundation
 2257 East 63 Street, Brooklyn,
New York 11234
 646-580-5610
…

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RAO Network Cycle 1 17
Natacha Pires (left), who was appointed as the first Executive Director of …

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18
Association for Creatine
Deficiencies
 6965 El Camino Real, Suite 105-598,
Carlsbad, CA …

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RAO Network Cycle 1 19
Heidi Wallis, Executive Director of the Association for Creatine
Deficien…

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20
The Champ Foundation
 4711 Hope Valley Road, 4F PMB 1171,
Durham, North Carolina 27707
 …

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RAO Network Cycle 1 21
The Champ Foundation hosted the SLSMDS Family and Scientific Conference at …

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22
CLOVES Syndrome
Community
 6716 Eastside Drive NE Ste 1-116,
Browns Point, Washington 98…

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RAO Network Cycle 1 23
Noreen Fairley, the CLOVES Syndrome Community’s Collaborative
Research Ne…

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24
Congenital Hyperinsulinism
International
 P.O. Box 135, Glen Ridge, New Jersey 07028
 97…

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RAO Network Cycle 1 25
An infant diagnosed with congenital hyperinsulinism.
• Through a detailed …

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26
Cure CMD
 3217 Carson St #306, Lakewood,
California 90712
 562-444-5656
 curecmd.org
…

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RAO Network Cycle 1 27
Mom and son share a common care regime for those living with
CMD: deliver…

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28
CureGRIN Foundation
 855-982-9470
 curegrin.org
To improve the lives of people around the…

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RAO Network Cycle 1 29
CureGRIN’s Meagan Hutchinson (right) presents Dr. Amy Ramsey
(left) with …

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30
Cure HHT
 P.O. Box 329, Monkton, Maryland 21111
 410-357-9932
 curehht.org
 hhtinfo@cu…

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RAO Network Cycle 1 31
Key publications
• Annals of Internal Medicine (2020): evidencebased cons…

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32
DADA2 Foundation
 7051 Hwy 70 South #353, Nashville,
Tennessee 37221
 615-647-8790
 da…

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RAO Network Cycle 1 33
Lex Cowsert, Chief Scientific Officer of DADA2 Foundation, speaks to fellow…

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34
DDX3X Foundation
 1000 N. West Street, Suite 900, Wilmington,
Delaware 19801
 304-997-95…

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RAO Network Cycle 1 35
DDX3X community members convene.
Key research and research infrastructure …

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36
The EHE Foundation
 1561 Hopi Court, Hobart, Wisconsin 54313
 877-460-4240
 fightehe.org…

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RAO Network Cycle 1 37
The EHE Foundation's former Executive Director Medha DeorasSutliff (right)…

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38
Fibrolamellar Cancer
Foundation
 20 Horseneck Lane, 2nd Floor, Greenwich,
Connecticut 06…

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RAO Network Cycle 1 39
• Identified and engaged high-potential early-career
investigators by fun…

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40
Glut1 Deficiency Foundation
 PO Box 737, Owingsville, Kentucky 40360
 859-585-2538
 g1df…

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RAO Network Cycle 1 41
Glut1 Deficiency Foundation team members and key research
collaborators g…

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42
Hermansky-Pudlak
Syndrome Network
 1 South Rd., Oyster Bay, New York 11771
 1-800-789-9H…

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RAO Network Cycle 1 43
Ashley Appell (left), of the Hermansky-Pudlak Syndrome Network,
connects …

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44
INADcure Foundation
 PO Box 11232, Fairfield, New Jersey 07004
 201-274–5204
 inadcure.o…

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RAO Network Cycle 1 45
Amanda Hope (left) and Leena Panwala (right), of the INADcure Foundation, c…

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46
KAT6 Foundation
 3 Louise Drive, West Nyack, New York 10994
 845-282-4979
 katfoundation…

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RAO Network Cycle 1 47
Angie Serrano (left) and Saylor Williams (right), of Boston
University, a…

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48
KIF1A.ORG
 1178 Broadway, New York, NY 10001
 kif1a.org
 impact@kif1a.org
To improve th…

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RAO Network Cycle 1 49
Families, researchers, and clinicians at the 2023 KAND Family and
Scienti…

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50
Lennox-Gastaut
Syndrome Foundation
 6030 Santo Road, Suite 1, Unit 420878,
San Diego, Ca…

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RAO Network Cycle 1 51
Tracy Dixon Salazar (center), Executive Director of the LGS
Foundation, c…

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52
Lymphangiomatosis &
Gorham's Disease Alliance
 7901 4th St. N STE 5761, St. Petersburg,
…

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RAO Network Cycle 1 53
Key research and research infrastructure achievements
• Tripled the size o…

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54
Li-Fraumeni Syndrome
Association
 PO Box 6458, Holliston, Massachusetts 01746
 833-469-5…

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RAO Network Cycle 1 55
Key research and research infrastructure achievements
• Jointly convened S…

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56
NEC Society
 140B St. Suite 5, #128, Davis, California 95616
 530-448-8088
 NECsociety.o…

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RAO Network Cycle 1 57
Members of the NEC Society community gather at the NEC
Symposium in 2023.…

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58
Project 8p Foundation,
of the Commission on
Novel Technologies for
Neurodevelopmental Cop…

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RAO Network Cycle 1 59
In summer 2021, Project 8p, Ring14 USA, and Dup15q Alliance
hosted the Mo…

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60
PSC Partners Seeking a Cure
 6900 E Belleview Ave Ste 202,
Greenwood Village, Colorado 801…

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RAO Network Cycle 1 61
PSC Partners Seeking a Cure staff attend the 2024 American
Association fo…

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62
Systemic JIA Foundation
 220 Valencia Drive, Los Altos, California 94022
 530-448-8088
 …

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RAO Network Cycle 1 63
Rashmi Sinha speaks during a session on patient communities as essential dr…

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64
The Snyder-Robinson
Foundation
 1443 Layman Street, McLean, Virginia 22101
 703-203-0215…

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RAO Network Cycle 1 65
Connor Raymond (center) and family visit with Cat Lutz, Ph.D. at The Jackso…

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66
TANGO2 Research
Foundation
 PO Box 43, Hadlyme, Connecticut 06439
 800-325-7391
 tango…

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RAO Network Cycle 1 67
Members of the TANGO2 team attend the Science in Society 2023 Meeting. Left…

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68
TESS Research Foundation
 PO Box 53, Menlo Park, California 94026
 650-521-2279
 tessres…

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RAO Network Cycle 1 69
Ellie (SLC13A5 Epilepsy Patient; center) with her family, Tanya
Brown, Ph…

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70
Usher 1F Collaborative
 321 Walnut Street, #228, Newtonville,
Massachusetts 02460
 339-22…

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RAO Network Cycle 1 71
The three children of Usher 1F Collaborative Founder and Board
President,…

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72
The Yaya Foundation for
4H Leukodystrophy
 5100 West 36th Street, #16404. St. Louis Park,
…

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RAO Network Cycle 1 73
Executive Director Ben Smith (right), and Jill Triptow (left), a
4HL pati…

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03 Conclusion 74
Making meaningful progress in rare disease requires
not only more research fund…

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chanzuckerberg.com
Building a Better
Future for Everyone

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@HeidiBP

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Rare As One Network Cycle 1 Impact Report

@HeidiBP

This impact report presents achievements from Cycle 1 of the Rare As One Network, a network of 94 patient-led rare disease organizations driving forward progress against rare disease. This report highlights the scientific momentum and progress these organizations have inspired, and the impact that a network of similarly placed and determined patient advocates can have when provided with baseline funding, capacity building support and opportunities to collaborate with one another.

Science

#RareAsOne #Patients #Research

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