About the Lysosomal Disease Network
www.lysosomaldiseasenetwork.org

More Information:
6702: Natural History and Structural Functional Relationships in Fabry Renal Disease
6703: Longitudinal Studies of Brain Structure and Function in MPS Disorders
6704: The Natural History of Mucolipidosis type IV
6706: A Historical Chart Review and Longitudinal Follow-Up of Identified Patients with Wolman Disease or Cholesteryl Ester Storage Disease, Lysosomal Acid Lipase Deficiency
6707: Characterizing the Neurobehavioral Phenotype(s) in MPS III (Pilot Study)
6708: Pulmonary Disease and Exercise Tolerance in Boys with Fabry Disease
6709: Longitudinal Follow-up of Individuals with Infantile Pompe Disease
6711: Expanded Screening for the Fabry Trait protocol
6713: A Natural History Study of the Gangliosidoses
6714: A Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I
6716: Genotype - Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis (2)
6718: Gene Therapy for Tay-Sachs Disease. Phase 1: Natural History Data Gather
6721: Intravenous N-acetylcysteine for the treatment of Gaucher's disease and Parkinson's disease
6724: Magnetic Resonance Spectroscopy (MRS) to Determine Neuroinflammation and Oxidative Stress in MPS I
6725: Podocyturia, a Non-Invasive Predictor of Renal Dysfunction in Fabry Nephropathy
6729: Synergistic Enteral Regimen for Treatment of the Gangliosidoses (Syner-G)
- Albert Einstein College of Medicine, Children 's Hospital at Montefiore, Bronx, New York City, NY
- Baylor Research Institute and Baylor College of Medicine, Dallas, TX
- Children's Hospital and Research Center Oakland, Oakland, CA
- Duke University, Durham, NC
- Emory University School of Medicine, Decatur, GA
- Hospital for Sick Children, Toronto, Ontario, Canada
- Joan and Sanford Weill Medical College of Cornell University, New York City, NY
- Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA
- Mayo Clinic, Rochester, MN
- New York University School of Medicine, New York City, NY
- State University of New York, University at Buffalo, Buffalo, NY
- University of California at San Francisco, San Francisco, CA
- University of Minnesota, Minneapolis, MN
- University of Washington, Seattle, WA
Chester B. Whitley, PhD, MD
Principal Investigator, Lysosomal Disease Network
Professor, Pediatrics
University of Minnesota Twin Cities
MMC 446 Mayo
420 Delaware Street, SE
Minneapolis, MN 55455
E-mail: whitley@umn.edu
David CC Erickson
Informatics Director, Lysosomal Disease Network
Phone: (612) 624-7975
E-mail: erick259@umn.edu
James C. Cloyd, PharmD
Co-Principal Investigator, Lysosomal Disease Network
Phone: (612) 624-4609
E-mail: cloyd001@umn.edu
Brenda Diethelm-Okita, MPA
Program Manager, Lysosomal Disease Network
Phone: (612) 625-1594
E-mail: dieth001@umn.edu
All inquiries about the LDN, LDN studies and participating sites, and LDN membership can be sent to info@lysosomaldiseasenetwork.org.
All Lysosomal conditions
- ANGEL AID
773 Center Blvd, Suite 464
Fairfax, CA 94930
415-662-8450
social@angelaidcares.org
OR text SIGNS to 741741 for the Crisis Text Line
Services are free and available 24/7.
ANGEL AID is a nurturing and safe community for mothers of children with rare disease. We offer relief- a place to be listened to without judgment, create relationships with women who truly understand, and learn from our network of health and wellness experts. All of this at no charge.
- Global Genes
Nicole Boice, Founder and CEO
nicoleb@globalgenes.org
- Lysosomal Diseases New Zealand
aka The New Zealand Lysosomal Storage Diseases Trust
John Forman, Chairperson
john.forman@xtra.co.nz
- Lysosomal Storage Disorders Support Society (“LSDSS”) (in India)
Shashank Tyagi, National Coordinator
shashanktyagi@lsdss.org
contactus@lsdss.org
- Rare Disorders NZ
Lisa Foster, Chief Executive
https://raredisorders.org.nz/contact-us/
- Quebec Coalition of Orphan Diseases
Jane Ricciardelli, COO
info@canadahelps.org
Alpha-mannosidosis and Aspartylglucosaminuria
- International Society for Mannosidosis and Related Diseases
www.ismrd.org
Jackie James, President;
Jenny Noble, Vice President & Administrator
jenny.noble@xtra.co.nz
167 Hollister Lane,
Ohauiti, Tauranga, New Zealand
Phone: 64 7 5448868
Cystinosis
- The Cystinosis Foundation
www.cystinosisfoundation.org
Valerie Hotz, Executive Director
VHotz@cystinosis.com
58 Miramonte Drive
Moraga, CA 94556
888-631-1588
- Cystinosis Research Foundation
www.cystinosisresearch.org
Nancy Stack
nstack@cystinosisresearch.org
Stacy Johnson
info@cystinosisresearch.org
sjohnson@cystinosisresearch.org
19200 Von Karman Ave, Suite 920
Irvine, CA 92612
949-223-7610
- Cystinosis Research Network
www.cystinosis.org
Christy Greeley, Executive Director
info@cystinosis.org
302 Whytegate Court
Lake Forest, IL 60045
866-276-3669
847-235-2773
Danon Disease
- Association for Glycogen Storage Disease
www.agsdus.orgIris Ferrecchia, President
irisferrecchia@gmail.com
P.O. Box 896
Durant, IA 52747
563-514-4022
Fabry Disease
- The Canadian Fabry Association
www.fabrycanada.com
Donna Strauss, President
donna_strauss@hotmail.com
Julia Alton, Vice President, Executive Director
altonjulia@gmail.com
748 Kelly Street
Thunder Bay, ON
Canada P7E 2A1
807-252-6388
- Fabry Australia, Inc.
www.fabry.com.au
Megan Fookes, OAM, Managing Director
director@fabry.com.au
secretary@fabry.com.au
P.O. Box 106, Mentone
Victoria, Australia 3194
61 (0)422 201 013
- Fabry Support and Information Group
www.fabry.org
Jack Johnson, Executive Director
JJohnson@fabry.org
PO Box 510
Concordia, MO 64020
660-463-1355
660-463-1356
- The National Fabry Disease Foundation
www.fabrydisease.org
Jerry Walter, Founder and President
jerry.walter@fabrydisease.org
info@fabrydisease.org
4301 Connecticut Ave. NW, Suite 404
Washington, DC 20008
800-651-9131
919-732-2799
Fucosidosis
- International Society for Mannosidosis and Related Diseases
www.ismrd.org
Jackie James, President;
Jenny Noble, Vice President & Administrator
jenny.noble@xtra.co.nz
167 Hollister Lane,
Ohauiti, Tauranga, New Zealand
Phone: 64 7 5448868
Gangliosidoses
- Cure GM1 Foundation
www.curegm1.org
Christine Waggoner, Founder
christinewaggoner@hotmail.com
christine@curegm1.org
P.O. Box 6890
Albany, CA 94706
- Cure Tay-Sachs Foundation
www.curetay-sachs.org
Rick Karl, President
lunchickm07@gmail.com
12730 Triskett Road
Cleveland, OH 44111
562-212-1071
- National Tay-Sachs and Allied Diseases Association (NTSAD)
www.ntsad.org
Susan Kahn, Executive Director
skahn@ntsad.org
2001 Beacon Street, Suite 204
Boston, MA 02135
800-906-8723
617-277-0134
-
New Hope Research Foundation
(for Adult-Onset Tay-Sachs disease)
www.newhoperesearch.org
Jack Keimel, President
jack.keimel@newhoperesearch.org
6 Charley Lake Court
North Oaks, MN 55127-6219
763-526-8064
Gaucher Disease
- Children's Gaucher Disease Research Fund
www.childrensgaucher.org
Gregory Macres, Founder and Chairman
research@childrensgaucher.org
PO Box 2123
Granite Bay, CA 95746
916-797-3700
916-797-3707
- Gaucher & LSD Society of South Africa
Kelly du Plessis, Executive Director
k.duplessis@mweb.co.za
- National Gaucher Foundation
www.gaucherdisease.org
Amy Blum, Chief Operating Officer
amy@gaucherdisease.org
Noreen Layne, Administrative and Development Assistant
noreen@gaucherdisease.org
5410 Edson Lane, Suite 220
Rockville, MD 20852
301-593-1452
800-504-3189
- National Gaucher Foundation of Canada
Christine White, President
christinewhite@gauchercanada.ca
christinewhite53@gmail.com
Glycogen Storage Diseases
- Association for Glycogen Storage Disease
www.agsdus.org
Iris Ferrecchia, President
irisferrecchia@gmail.com
P.O. Box 896
Durant, IA 52747
563-514-4022
Krabbe Disease
- Hunter's Hope Foundation
www.huntershope.org
Jacque Waggoner, Chief Executive Officer
jacque@huntershope.org
info@huntershope.org
anna@huntershope.org
6368 West Quaker Street
Orchard Park, NY 14127
877-984-4673 toll free
716-667-1200
716-667-1212 fax - KrabbeConnect
www.KrabbeConnect.org
Stacy Pike-Langenfeld, Co-Founder and President
stacy.pike@krabbeconnect.org
Anne Rugari, Co-Founder and Vice-President
anne.rugari@krabbeconnect.org
P.O. Box 2643
Rosemount, MN 55068-0264
651-252-4117 - The Legacy of Angels Foundation
(For Krabbe disease & cystic fibrosis)
www.tloaf.org
Paul Rosenau, President/Co-Founder
stacyluken@icloud.com
Stacy Pike-Langenfeld, Director of Programs and Administration
stacypike@tloaf.org
P.O. Box 1014
Prior Lake, MN 55372
612-387-3424
Leukodystrophies
- Adrenoleukodystrophy Foundation
www.aldfoundation.org
David A. Cry, CEO
information@aldfoundation.org
241 Camden Street
Slidell, LA 70461
985-718-4728
- Australasian Leukodystrophy Foundation
Bob Wyborn, President and Founder
BobWyborn@bigpond.com
- Chloe’s Fight Rare Disease Foundation
(For metachromatic leukodystrophy)
www.chloesfight.org
Erica Barnes, Co-Founder and Chair of the Board of Directors
erica.barnes@rareaction.org
Erica.Barnes@rarediseases.org
ericademourebarnes@gmail.com
343 Sweet Briar Lane
Hopkins, MN 55343
952-457-6956
- MLD Foundation
(For metachromatic leukodystrophy)
www.mldfoundation.org
Teryn Suhr, Executive Director
teryn@mldfoundation.org
Dean Suhr, President & Board Chairperson
dean@mldfoundation.org
MLDinfo@mldfoundation.org
21345 Miles Drive,
West Linn, OR 97068
800-617-8387
503-656-4808
- MLD Support Association UK
(for metachromatic leukodystrophy)
Vivienne Clark, Chairperson
Jackie Imrie, Trustee
admin@mldsupportuk.org.uk
- United Leukodystrophy Foundation
www.ulf.org
Bobbi Burgstone, Executive Director
bobbi@ulf.org
224 North Second Street, Suite 2
DeKalb, IL 60115
800-728-5483 or (815) 748-3211
Fax: (815) 748-0844
Mucolipidosis I (Sialidosis)
- International Society for Mannosidosis and Related Diseases
www.ismrd.org
Jackie James, President;
Jenny Noble, Vice President & Administrator
jenny.noble@xtra.co.nz
167 Hollister Lane,
Ohauiti, Tauranga, New Zealand
Phone: 64 7 5448868
Mucolipidosis II/III (I-cell and pseudo-Hurler Polydystrophy)
- International Society for Mannosidosis and Related Diseases
www.ismrd.org
Jackie James, President;
Jenny Noble, Vice President & Administrator
jenny.noble@xtra.co.nz
167 Hollister Lane,
Ohauiti, Tauranga, New Zealand
Phone: 64 7 5448868
Mucolipidosis IV
- Mucolipidosis IV Foundation - ML4 Foundation
www.ml4.org
Rebecca Oberman, Ph.D., Executive Director
roberman@ml4.org
info@ml4.org
3500 Piedmont Road, Suite 500
Atlanta, GA 30305
404-510-2577
Mucopolysaccharidosis (MPS)
- Ben's Dream
Sanfilippo Research Foundation
www.bensdream.org
Stuart & Jennifer Siedman
P.O. Box 81268
Wellesley, MA 02481-0002
617-899-4158
- The Canadian MPS Society (The Canadian Society for Mucopolysaccharide & Related Diseases Inc.)
www.mpssociety.ca
Kimberly D. Angel, Executive Director
kimangel@mpssociety.ca
#218-2055 Commercial Drive
Vancouver, BC V5N 0C7
Canada
1-800-667-1846 or 604-924-5130
- Cure Sanfilippo Foundation
www.curesff.org
Glenn O'Neill, President
curesff@gmail.com
Dr. Cara O'Neill, MD, Scientific Director
cara.curesff@gmail.com
P.O. Box 6901
Columbia, SC 29260
803-413-0525
- The Isaac Foundation
(For MPS diseases)
www.theisaacfoundation.com
Alexandra Hall, Managing Director; Policy, Patient Support, & Industry Liaison
alix@theisaacfoundation.com
mcfadyena@me.com
5291 County Rd. 30
Campbellford, Ontario
Canada K0L 1L0
613-328-9136
Twitter: @isaacfoundation
YouTube: www.youtube.com/user/mcfadyena
-
Jonah's Just Begun - Foundation to Cure Sanfilippo
www.jonahsjustbegun.org
Jeremy Weishaar, President
Jill Wood, Treasurer
P.O. Box 150057
Brooklyn, NY 11215
- National MPS Society
www.mpssociety.org
Terri Klein, President and CEO
Terri@mpssociety.org
1007 Slater Rd, Suite 220
Durham, NC 27703
919-806-0101
919-806-2055
- Project Alive
(for Hunter Syndrome)
www.projectalive.org
Melissa Hogan, JD, President
melissa.hogan@projectalive.org
give@projectalive.org
P.O. Box 384
Thompson's Station, TN 37179
313-312-5483
- The Ryan Foundation
www.ryanfoundation.net/home
Mark Dant, Executive Director
info@ryanfoundation.org
2025 Jackson Rd
Carrollton, Texas 75006-1739
214-870-7926
- The Sanfilippo Children's Foundation
(an Australian PAG)
www.sanfilippo.org.au
Megan Donnell, Executive Director
megan.donnell@sanfilippo.org.au
P.O. Box 475, Freshwater
NSW 2096, Australia
1800 664 878
- Sanfilippo Foundation for Children
www.sf4k.org
Roy Zeighami, Executive Director/Founder
6420 Diamond Dr
Mckinney, TX 75070
- The Society for Mucopolysaccharide Diseases (UK)
Bob Stevens, Group Chief Executive
info@mpssociety.org.uk
- Taiwan MPS Society
Virginia Tsai, Founder
tsaivirginia@yahoo.com.tw
Megan Sun, Co-Admin
taiwanmpssociety@gmail.com
Multiple Sulfatase Deficiency
- MSD Action Foundation
(for multiple sulfatase deficiency)
www.savingdylan/about-the-foundation.com
Alan Finglas
alanfinglas@gmail.com
MSD Action Foundation
c/o Grattan Lodge
Balgriffin, Dublin 13
Ireland
- United MSD Foundation, Inc.
(for multiple sulfatase deficiency)
www.unitedmsdfoundation.com
Amber Olsen
WarriorsForWillow@gmail.com
aolsen@nextaff.com
2112 Bienville Blvd, Suite B2
Ocean Springs, MS 39564
228-327-6916
Neuronal Ceroid Lipofuscinosis (Batten disease)
- Batten Disease Support and Research Association
www.bdsra.org
Tracy Kirby, BDSRA Family Liaison
tkirby@bdsra.org
Noreen Murphy, Admin
nmurphy@bdsra.org
2780 Airport Dr, Suite 342
Columbus, OH 43219-2292
800-448-4570
- The Charlotte and Gwenyth Gray Foundation
www.curebatten.org
Kristen and Gordon Gray (the parents)
c/o The Giving Back Fund
curebatten@givingback.org
graygirlsfoundation@gmail.com
5757 W. Century Blvd, Suite 410
Los Angeles, CA, 90045
310-649-5222
- Nathan's Battle Foundation (for LINCL)
www.nathansbattle.com
Phil Milto, Executive Director
pmilto@indy.net
459 State Road 135
South Greenwood, IN 46142
317-888-7396
317-888-0504
- Taylor's Tale
(Originally for Batten disease; now for all rare diseases)
www.taylorstale.org
Laura King Edwards, Co-founder and Vice President
laura@taylorstale.org
Sharon King, President
sharon@taylorstale.org
7804 Fairview Rd, Box 135
Charlotte, NC 28226-4998
Niemann-Pick Disease
-
Addi & Cassi Fund
(For Niemann-Pick disease type C)
www.addiandcassi.com
Hugh Hempel and Chris Hempel
59 Damonte Ranch Parkway
Suite B360
Reno, NV 89511
800-517-2542
- The Ara Parseghian Medical Research Foundation
www.parseghianfund.nd.edu
Sean Kassen, Ph.D., Director of the Ara Parseghian Medical Research Fund at the University of Notre Dame
skassen@nd.edu
Ara Parseghian Medical Research Fund
University of Notre Dame
Office of the Dean
215 Jordan Hall of Science
Notre Dame, IN 46556-4618
574-631-4689
- Hide & Seek Foundation/SOAR
(For Niemann-Pick type C)
www.hideandseek.org
Jonathan Jacoby, President
6475 Pacific Coast Highway, Suite 466
Long Beach, CA 90803
844-762-7672
- National Niemann-Pick Disease Foundation
www.nnpdf.org
Joslyn Crowe, Executive Director
jcrowe@nnpdf.org
PO Box 49
Fort Atkinson, WI 53538
920-563-0930
877-287-3672
- Niemann-Pick UK (NPUK)
Toni Mathieson, Chief Executive
toni@npuk.org
- The Quinn Madeleine Foundation
www.quinnmadeleine.org
Eileen Linzer, Co-Founder and Executive Director
eileen.linzer@quinnmadeleine.org
PO Box 72
Lynbrook, NY 11563
516-206-2155
Pompe Disease
- Acid Maltase Deficiency Association
www.amda-pompe.org
Tiffany House, AMDA President
TiffanyLHouse@aol.com
PO Box 700248
San Antonio, TX 78270
210-494-6144
210-490-7161
- Australian Pompe's Association, Inc.
www.australianpompe.org
Raymond Saich, President
RSaich@bigpond.net.au
8 Follett Rd
Cheltenham, Victoria 3192
Australia
- Canadian Association of Pompe,
a.k.a. Association Canadienne de Pompe
www.pompecanada.com
Brad Crittenden, President
brad@pompecanada.com
112-3201 Wilson Street
Penticton, BC
Canada V2A 8J3
- United Pompe Foundation
David W. Hamlin
david@unitedpompe.com
Salla disease
- Salla Treatment and Research Foundation
Jessica Klein-Foglio, President
jessica@starforben.org
Michael Foglio
mike@starforben.org
Schindler disease
- International Society for Mannosidosis and Related Diseases
www.ismrd.org
Jackie James, President;
Jenny Noble, Vice President & Administrator
jenny.noble@xtra.co.nz
167 Hollister Lane,
Ohauiti, Tauranga, New Zealand
Phone: 64 7 5448868
Wolman Disease
-
LAL Solace
www.lalsolace.org
Brett Billmeyer, Executive Director
Mnwild11@aol.com
11478 Sawmill Curve
Woodbury, MN 55129