Lysosomal Diseases

Advocacy & Support Groups

★★These patient advocacy groups provided formal letters of support for the Lysosomal Disease Network’s 2014 NIH grant application.

※※These patient advocacy groups provided formal letters of support for the Lysosomal Disease Network’s 2019 NIH grant application.

❖These patient advocacy groups are affiliated with the Lysosomal Disease Network.

North America

Acid Maltase Deficiency Association (AMDA)
Tiffany House, President
P.O. Box 700248-0248
San Antonio, Texas 78270, USA
Tel. (210) 494-6144
Email: AMDA President

Addi & Cassi Fund
(For Niemann-Pick disease type C)
59 Damonte Ranch Parkway
Suite B360
Reno, NV 89511, USA
Tel. (800) 517-2542
Website: Addi & Cassi Fund

Adrenoleukodystrophy Foundation
David A. Cry, CEO
241 Camden Street
Slidell, LA 70461, USA
Tel. (985) 718-4728

The Ara Parseghian Medical Research Foundation
(For Niemann-Pick disease type C)
Sean Kassen, PhD, Director
The Ara Parseghian Medical Research Fund
University of Notre Dame
Office of the Dean
215 Jordan Hall of Science
Notre Dame, IN 46556-4618, USA
Tel (574) 631-4689
Email: Director of the Ara Parseghian Medical Research Foundation
Website: The Ara Parseghian Medical Research Foundation

Association for Glycogen Storage Disease (AGSD)
Iris Ferrecchia, President
P.O. Box 896
Durant, IA 52747, USA
Tel. (563) 514-4022
Email: President of the AGSD

※※❖Batten Disease Support and Research Association (BDSRA)
Tracy Kirby, BDSRA Family Liaison
2780 Airport Drive, Suite 342
Columbus, OH 43219-2292, USA
Tel. (800) 448-4570
Email: BDSRA Family Liaison

Ben’s Dream Sanfilippo Research Foundation
Stuart & Jennifer Siedman
P.O. Box 81268
Wellesley, MA 02481-0002, USA
Tel. (617) 899-4158

Canadian Association of Pompe
(Association Canadienne de Pompe)
Brad Crittenden, President
112-3201 Wilson Street
Penticton, BC
Canada V2A 8J3
Email: Brad Crittenden

Canadian Fabry Association
Gina Costantino, President
Donna Strauss, Executive Director
52 Glen Forest Drive
Hamilton, ON
Canada L8K 5V8
Email: Executive Director of the Canadian Fabry Association

※※★★❖The Canadian MPS Society (The Canadian Society for Mucopolysaccharide & Related Diseases Inc.)
Kimberly D. Angel, Executive Director
#218-2055 Commercial Drive
Vancouver, BC
Canada V5N 0C7
Tel. (800) 667-1846 or (604) 924-5130
Email: Executive Director of the Canadian MPS Society

The Charlotte and Gwenyth Gray Foundation
c/o The Giving Back Fund
5757 W. Century Blvd., Suite 410
Los Angeles, CA 90045, USA
Tel. (310) 649-5222
Email: The Charlotte & Gwenyth Gray Foundation

Children’s Gaucher Research Fund
Greg Macres
8110 Warren Court
Granite Bay, CA 95746, USA
Tel. (916) 797-3700

※※❖Chloe’s Fight Rare Disease Foundation
(For metachromatic leukodystrophy)
Erica Barnes, Co-founder & Chair of the Board of Directors
343 Sweet Briar Lane
Hopkins, MN 55343, USA
Tel. (952) 457-6956
Email: Chloe’s Fight Rare Disease Foundation
Website: Chloe’s Fight

Cure GM1 Foundation
Christine Waggoner, Founder
P.O. Box 6890
Albany, CA 94706, USA
Email: Christine Waggoner
Website: Cure GM1 Foundation

※※❖Cure Sanfilippo Foundation
Glenn O’Neill, President
P.O. Box 6901
Columbia, SC 29260, USA
Tel. (803) 413-0525
Email: Glenn O’Neill
Website: Cure Sanfilippo Foundation

Cure Tay-Sachs Foundation
Rick Karl, President
12730 Triskett Road
Cleveland, OH 44111, USA
Tel. (562) 212-1071
Email: Rick Karl
Website: Cure Tay-Sachs Foundation

The Cystinosis Foundation
Valerie Hotz, Executive Director
58 Miramonte Drive
Moraga, CA 94556, USA
Tel. (888) 631-1588 or (925) 631-1588
Email: Cystinosis Foundation Executive Director

Cystinosis Research Foundation
Nancy Stack, President and Founding Trustee
18802 Bardeen Avenue
Irvine, CA 92612, USA
Tel. (949) 223-7610
Email: Contact the Cystinosis Research Foundation
Website: Cystinosis Research Foundation

Cystinosis Research Network
Christy Greeley, Executive Director
302 Whytegate Court
Lake Forest, IL 60045, USA
Tel. (847) 735-0471 or (866) 276-3669

※※★★❖Fabry Support and Information Group
Jack Johnson, Founder and Executive Director
108 NE 2nd Street, Suite C
P.O. Box 510
Concordia, MO 64020, USA
Tel. (660) 463-1355, Fax: (660) 463-1356
Email: Fabry Support & Information Group Executive Director

Hide and Seek Foundation
(For Niemann-Pick disease type C)
Jonathan Jacoby, President
6475 East Pacific Coast Highway, Suite 466
Long Beach, CA 90803, USA
Tel. (844) 762-7672
Email: none available and no online contact form

※※★★❖Hunter’s Hope Foundation
(For Krabbe disease)
Jacque Waggoner, Chief Executive Officer
6368 West Quaker Street
P.O. Box 643
Orchard Park, NY 14127, USA
Tel. (716) 667-1200
Website: Hunter’s Hope Foundation

The International Niemann–Pick Disease Alliance (INPDA)
The INPDA’s office is located in England. The INPDA is a global network that includes not-for-profit member Niemann–Pick disease patient advocacy associations from England and Wales, Argentina, Australia, Brazil, Canada, Republic of China (Taiwan), France, Germany, Italy, the Netherlands, Norway, Scotland, Spain, Switzerland, and the United States. The INPDA convenes regular teleconferences, and also holds a biennial face-to-face conference.
Tel. +44 (0)191 4150693
Website: International Niemann–Pick Disease Alliance

The Isaac Foundation
(For MPS diseases)
Alexandra Hall, Managing Director
5291 County Rd. 30
Campbellford, Ontario
Canada K0L 1L0
Tel. (613) 328-9136
Email: Alexandra Hall
Website: The Isaac Foundation

Jonah’s Just Begun – Foundation to Cure Sanfilippo, Inc.
(For Sanfilippo syndrome type C, a.k.a. MPS IIIC)
Jeremy Weishaar, President
Jill Wood, Treasurer
P.O. Box 150057
Brooklyn, NY 11215, USA
Tel. (347) 831-0246
Email for Patient Outreach: Jill Wood
Email for All Other Inquiries: Jonah’s Just Begun Admin
Websites: Jonah’s Just Begun
Jonah’s Just Begun on FaceBook

Stacy Pike-Langenfeld, Co-Founder and President
Anne Rugari, Co-Founder and Vice-President
P.O. Box 264
Rosemount, MN 55068-0264, USA
Tel. (651) 252-4117
Website: KrabbeConnect

LAL Solace (Support Organization for LAL deficiency – Advocacy, Care and Expertise)
(For lysosomal acid lipase deficiency, a.k.a. LALD / Wolman disease / cholesteryl ester storage disease (CESD))
Brett Billmeyer, Executive Director
Minnesota, USA
Email: Brett Billmeyer at LAL Solace

※※❖The Legacy of Angels Foundation
(For Krabbe disease & cystic fibrosis)
(The Legacy of Angels Foundation is a private grant-making organization with a
preference for leveraged, strategic funding. Applications are by invitation only.)
Paul Rosenau, President and Co-Founder
Stacy Pike-Langenfeld, Director of Programs and Administration
P.O. Box 1014
Prior Lake, MN 55372, USA
Email: Stacy Pike
Website: The Legacy of Angels Foundation

Little People of America, Inc.
(This group includes persons who have mucopolysaccharidosis IVA/MPS IVA/Morquio syndrome type A. It has very active regional chapters throughout the United States, as well as national-level events, programs and advocacy.)
Mark Povinelli, President
617 Broadway, #518
Sonoma, CA 95476, USA
Tel. Voice: (714) 368-3689 or toll-free (888) LPA-2001; Fax: (707) 721-1896
Website: Little People of America, Inc.

※※★★❖MLD Foundation
(For metachromatic leukodystrophy)
Teryn Suhr, Executive Director; Dean Suhr, President & Board Chairperson
21345 Miles Drive
West Linn, OR 97068-2878, USA
Tel. (800) 617-8387 or (503) 656-4808
Website: MLD Foundation

★★❖Mucolipidosis Type IV Foundation (ML4 Foundation)
Rebecca Oberman, PhD, Executive Director
3500 Piedmont RD, Suite 500
Atlanta, GA 30305, USA
Tel. (877) 654-5459

National Fabry Disease Foundation
Jerry Walter, Founder and President
4301 Connecticut Ave. N.W.
Suite 404
Washington D.C. 20008, USA
Tel. (800) 651-9131

★★❖National Gaucher Foundation
Amy Blum, Executive Director
5410 Edson Lane, #220
Rockville, MD 20852, USA
Tel. (800) 504-3189
Email: National Gaucher Foundation

※※❖National Gaucher Foundation of Canada
Christine White, President
83 Winnegreen Court
Ottawa, Ontario
Canada K1G 5S3
Tel. (613) 867-6344
Email: National Gaucher Foundation of Canada President

※※★★❖National MPS Society
Terri Klein, President and CEO
1007 Slater RD, Suite 220
Durham, NC 27703, USA
Tel. (919) 806-0101,  Fax: (919) 806-2055
Email: National MPS Society

National Niemann-Pick Disease Foundation
Joslyn Crowe, Executive Director
P.O. Box 49
401 Madison Ave, Ste. B
Ft. Atkinson, WI 53538, USA
Tel. (920) 563-0930 or (877) 287-3672, Fax (920) 563-0931

※※★★❖National Tay-Sachs and Allied Diseases Association Inc.
Susan Kahn, Executive Director
2001 Beacon Street, Suite 204
Boston, MA 02135, USA
Tel. (617) 277-4463, Fax (617) 277-0134

New Hope Research Foundation
(For adult-onset Tay-Sachs disease)
Jack Keimel, President
6 Charley Lake Court
North Oaks, MN 55127-6219, USA
Tel. (763) 526-8064

New Jersey Rare Disease Alliance
W.E. “Bill” Cummings, Jr., Committee Member
New Jersey, USA
Tel. (781) 249-9773
Email: New Jersey Rare Disease Alliance Admin
Website: none

Project Alive
(For MPS II / Hunter syndrome)
Melissa Hogan, JD, President
P.O. Box 384
Thompson’s Station, TN 37179, USA
Tel. (313) 312-5483
Email: Contact Project Alive

Quebec Coalition of Orphan Diseases
Regroupement québécois des maladies orphelines (RQMO)
Gail Ouellette, PhD, Executive Director
110, avenue des Pins Ouest
Montréal (Québec)
Canada H2W 1R7
Tel. (819) 543-0550

The Quinn Madeleine Foundation
(For Niemann-Pick disease type A (NPA))
Eileen Linzer, Co-Founder & Executive Director
Quinn Madeleine Inc.
P.O. Box 721
Lynbrook, NY 11563, USA
Tel. (516) 206-2155
Email: Executive Director of the Quinn Madeleine Foundation
Website: The Quinn Madeleine Foundation

★★❖The Ryan Foundation
(For the mucopolysaccharidoses (MPS diseases) and other lysosomal diseases)
Mark Dant, Executive Director
2025 Jackson Road
Carrollton, TX 75006, USA
Tel. Voice: (214) 870-7926 or Fax: (972) 466-3522
Email: The Ryan Foundation Executive Director or submit your message on their Contact Form
Website: The Ryan Foundation

Salla Treatment and Research Foundation (“A STAR for Ben, Inc.”)
Jessica and Michael Foglio, Co-Founders
P.O. Box 1051
Riverdale Station
Bronx, NY 10471, USA
Tel. (347) 843-0907

Sanfilippo Foundation for Children
Roy Zeighami, Executive Director/Founder
6420 Diamond DR
Mckinney, TX 75070, USA

※※❖Taylor’s Tale
(For Batten disease, and rare diseases as a whole)
Sharon King, President
7804 Fairview Road, Box 135
Charlotte, NC 28226-4998, USA
Email: Sharon King
Website: Taylor’s Tale

Team Sanfilippo Foundation (TSF Inc.)
Kathleen Buckley, President
Patty Taormino, Vice President
20 Lakewood Avenue
Ronkonkoma, NY 11779, USA
Tel. (410) 227-5711
Email: Team Sanfilippo Foundation V.P. or Team Sanfilippo Foundation President
Websites: Team Sanfilippo Foundation and Team Sanfilippo Foundation on Facebook

United Leukodystrophy Foundation
Bobbi Burgstone, Executive Director
224 North Second Street, Suite 2
DeKalb, IL 60115-3293, USA
Tel. (800) 728-5483 or (815) 748-3211
Email: United Leukodystrophy Foundation Executive Director
Website: United Leukodystrophy Foundation

United MSD Foundation, Inc.
(For multiple sulfatase deficiency)
Amber Olsen
P.O. Box 929
Ocean Springs, MS 39566-9982, USA
Tel. (228) 327-6916
Email: United MSD Foundation
Website: Warriors for Willow / United MSD Foundation

★★❖United Pompe Foundation
David W. Hamlin
5100 N. Sixth Street, #119
Fresno, CA 93710, USA
Tel. (559) 227-1898, Fax (559) 227-1898
Email: United Pompe Foundation Admin

★★These patient advocacy groups provided formal letters of support for the Lysosomal Disease Network’s 2014 NIH grant application.

※※These patient advocacy groups provided formal letters of support for the Lysosomal Disease Network’s 2019 NIH grant application.

❖These patient advocacy groups are affiliated with the Lysosomal Disease Network.

The listing of any external web page on the Lysosomal Disease Network web site does not imply or express any endorsement or guarantee of the information or hyperlinks presented on that external web page.


Lysosomal Storage Disorders Support Society (“LSDSS”) (in India)
Multiple Contacts Throughout India:
Shashank Tyagi, National Coordinator
Dept. of Genetic Medicine,
Ground Floor – near Emergency Block
Sir Gangaram Hospital, Old Rajender Nagar,
New Delhi – 110065
Tel. +91 98100 22645
Websites: and

Taiwan MPS Society
Virginia Tsai, Founder
7F, No. 16, Ln. 102, Hejiang St.
Zhongshan Dist.
Taipei City 104, Taiwan (R.O.C.)
Email: Information Requests

Australia and New Zealand

Australasian Leukodystrophy Foundation
Bob Wyborn, President and Founder
P.O. Box 209
Clontarf Beach 4019
Tel. 61-7-3283 7224
Email: Bob Wyborn
Website: Australasian Leukodystrophy Foundation

Australian Battens Support Group
Harry Partridge, President
9 Norton Avenue
Killarney Vale
NSW 2261

The Australian MPS Society (Mucopolysaccharide & Related Diseases Society Aust. Ltd.)
Vanessa Ede-Scott, Board President
P.O. Box 131
Heidelberg, VIC 3084
Tel. 1800 045 515

Australian Pompe’s Association, Incorporated
Raymond Saich, President
8 Follett Rd
Cheltenham, Victoria 3192
Tel. +61 02 9629 2842
Email: President of the Australian Pompe’s Association
Website: Australian Pompe’s Association

※※❖Fabry Australia Inc.
Megan Fookes, Managing Director
P.O. Box 106
Mentone, Victoria, Australia 3194
Tel. +61 (0)422 201 013
Website: Fabry Australia

ISMRD — International Advocate for Glycoprotein Storage Diseases
(International Society of Mannosidosis and Related Diseases)
Jenny Noble, Vice President & Administrator
167 Hollister Lane
Ohauiti, Tauranga
New Zealand
Tel. 64 7 5448868 / 64 021 548318
Email: Jenny Noble
Website: ISMRD

Leukodystrophy Resource and Research Organisation Inc.
Bob Wyborn, President and Founder
P.O. Box 209
Clontarf Beach 4019
Tel. 61-7-3283 7224
Email: Bob Wyborn
Website: Leukodystrophy Resource and Research Organisation Inc.

Lysosomal Diseases New Zealand (The New Zealand Lysosomal Storage Diseases Trust)
John Forman, Chairperson
125 Cuba Street
Lower Hutt City
New Zealand
Tel. 04 566 7707
Email: Lysosomal Diseases New Zealand Chairperson

New Zealand Organisation for Rare Disorders (NZORD)
Letitia O’Dwyer, Chief Executive
P.O. Box 38-538
Wellington Mail Centre
Wellington, New Zealand 5045
Tel. 64 4 471 2226

※※❖Sanfilippo Children’s Foundation
P.O. Box 475
Freshwater NSW 2096
Tel. 1800 664 878


European Gaucher Alliance
Tanya Collin-Histed, CEO
Administrative Address:
8 Silver Street
Gloucestershire, GL11 4ND
United Kingdom
Tel/Fax: 00 (44) 1453 796402
Online Contact Form:
Website: European Gaucher Alliance

Fabry International Network
Jack Johnson, Acting President
Lut De Baere, Acting Vice President
Administrative location: Amersham, United Kingdom

Genetic & Rare Disorders Organisation (in Ireland)
C/O Fighting Blindness
3rd Floor, 7 Ely Place
Dublin 2, Ireland
Tel. 086 0229262
Website: Genetic & Rare Disorders Organisation

※※❖MSD Action Foundation (in Ireland)
(For multiple sulfatase deficiency)
Alan Finglas, Co-Founder
MSD Action Foundation
c/o Grattan Lodge
Balgriffin, Dublin 13, Ireland
Email: Information at MSD Action Foundation
Website: MSD Action Foundation

German MPS Society (Gesellschaft für Mukopolysaccharidosen e.V.)
Herstallstraße 35
63739 Aschaffenburg
Tel. 0 60 21 – 85 83 73, Fax 0 60 21 – 85 83 72
Website: Gesellschaft für Mukopolysaccharidosen e.V., in German

German NCL (Batten Disease) Society (NCL-Gruppe Deutschland e.V.)
NCL-Gruppe Deutschland e.V.
Iris Dyck, Chairperson
Hermann-Piper-Straße 8
13403 Berlin
Tel. 030 / 411 26 19
E-mail: German NCL Society Chairperson
Website: NCL-Gruppe Deutschland e.V., in German

German Pompe Disease Society (Pompe Deutschland e.V.)
Thomas Schaller, Chairman
Höhefeldstr. 26
76356 Weingarten
Tel. 49 (0)7244 1230
Website: Pompe Deutschland e.V., in German

Italian MPS Society (Associazione italiana mucopolisaccaridosi e malattie affini – Onlus)
Flavio Bertoglio, President
Via Volta, 291 – 20030 Senago (MI)
Milan, Italy
Tel. 39 02.99010188, Fax 39 02.99486016

French Lysosomal Diseases Society (Vaincre Les Maladies Lysosomales)
2 ter avenue de France
91300 MASSY
Tel. 01 69 75 40 30, Fax 01 69 36 93 50
Website:, in French

South Africa

Gaucher and LSD Society of South Africa (GLSDSA)
Kelly du Plessis, Executive Director
Juile 37, Private Bag X2
Danfern, Johannesburg 2055
South Africa
Email: Gaucher & LSD Society of South Africa Executive Director
Website: GLSDSA on FaceBook

South America

Brazilian MPS Association (APMPS – Associação Paulista de Mucopolissacaridoses)
Regina Próspero, President
Located in São Paulo, Brasil
Tel. (11) 2414-3060 or (11) 97385-0347
Website: Instituto Vidas Raras

United Kingdom

The Association for Glycogen Storage Disease (UK) Limited (AGSD-UK)
Michael Porter, Chairperson
Correspondence Address:
Old Hambledon Racecourse, Sheardley Lane
Droxford, SO32 3QY
United Kingdom
Tel. 0300 123 2790 and 0300 123 2799
Email: Association for Glycogen Storage Disease (UK) Chairperson

Batten Disease Family Association (BDFA)
Amanda Mortensen, Board Chair
209-211 City Road
London, EC1V 1JN
United Kingdom
Tel. 07876 682589 for general inquiries
Email: Board Chair

CLIMB – Children Living with Inherited Metabolic Diseases
Climb Building
176 Nantwich Road, Crewe
Cheshire CW2 6NG
United Kingdom
Tel. 0845 241 2173, Fax 01270 250244
Website: temporarily out-of-service

MLD Support Association UK
(For metachromatic leukodystrophy)
Jackie Imrie
Floor 5, Amphenol Business Complex
Thanet Way, Whitstable
United Kingdom CT5 3SB
Tel. 07836 627485 / 07977 440809
Website: MLD Support Association UK

Niemann-Pick UK (NPUK)
Toni Mathieson, Chief Executive
Suite 2, Vermont House, Concord, Washington
Tyne and Wear, NE37 2SQ
United Kingdom
Tel. 0191 415 0693

The Society for Mucopolysaccharide Diseases (UK)
Bob Stevens, Group Chief Executive
MPS House, Repton Place, White Lion Road
Amersham, Buckinghamshire, HP7 9LP
United Kingdom
Tel. 0345 389 9901, Fax 0345 389 9902

The listing of any external web page on the Lysosomal Disease Network web site does not imply or express any endorsement or guarantee of the information or hyperlinks presented on that external web page.

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