2022 Exhibitors & Patient Advocate Showcase

Batten Disease Support and Research Association

Cure GM1 Foundation

Cure Sanfilippo Foundation

Global Genes

The International Working Group on Gaucher Disease (IWGGD)

Living in the Light

MLD Foundation

National MPS Society

NORD

NTSAD (National Tay-Sachs & Allied Diseases Association)

Amicus Therapeutics

4DMT

Aceragen

Aeglea BioTherapeutics

AllStripes Research Inc.

Amicus Therapeutics

ARCHIMEDlife

Astellas Gene Therapies

AVROBIO

Azafaros

BioMarin Pharmaceutical Inc.

Chiesi Global Rare Diseases

Denali Therapeutics

European GD Network Charitable Foundation

Freeline Therapeutics Ltd

Greenwood Genetic Center

Homology Medicines, Inc.

Idorsia Pharmaceuticals Ltd

Igenomix

JCR Pharmaceuticals Co., Ltd.

Lysosomal & Rare Disorder Research & Treatment Center

M6P Therapeutics (M6PT)

Neurogene Inc.

Orchard Therapeutics

Orphazyme A/S

Paradigm Biopharmaceuticals

Passage Bio

Pfizer

Rare Disease Data Trust (RDDT)

REGENXBIO Inc.

Sanofi Genzyme

SEI Healthcare

Sigilon Therapeutics

Spark Therapeutics

Takeda Pharmaceuticals

Taysha Gene Therapies

Travere Therapeutics

Ultragenyx Pharmaceutical Inc.

Batten Disease Support and Research Association

Cure GM1 Foundation

Cure Sanfilippo Foundation

Fabry International Network

National MPS Society

United MSD Foundation

Amicus Therapeutics

AskBio

Audentes Therapeutics, an Astellas Company

BioMarin Pharmaceutical

CENTOGENE GmbH

Chiesi Global Rare Diseases

Denali Therapeutics, Inc.

Freeline

Forge Biologics

Homology Medicines, Inc.

Idorsia Pharmaceutical

Invitae

JCR Pharmaceuticals Co., Ltd.

Orchard Therapeutics

Orphazyme

Passage Bio

Pfizer

QPS Austria GmbH

Sanofi Genzyme

Sigilon Therapeutics

Spark Therapeutics

Takeda

Travere Therapeutics

Ultragenyx Pharmaceutical Inc.

Batten Disease Support and Research Association

Cure Sanfilippo Foundation

European Working Group on Gaucher Disease

EveryLife Foundation for Rare Diseases

Fabry International Network

Global Genes

MLD Foundation

MSD Action Foundation

National Fabry Disease Foundation

National MPS Society

Salla Treatment and Research Foundation

United MSD Foundation

Amicus Therapeutics, Inc.

ARCHIMEDlife

Audentes Therapeutics

Baebies

BioMarin Pharmaceutical

Blueprint Genetics

CENTOGENE US LLC

Charles River

Chiesi Farmaceutici S.p.A.

Denali Therapeutics

Enzyvant

European GD Network

Greenwood Genetic Center

Homology Medicines, Inc.

Idorsia Pharmaceutical

Invitae

JCR Pharmaceuticals Co., Ltd.

Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)

Neurogene Inc.

Orchard Therapeutics

Orphazyme

Patient Discovery

Pfizer, Inc.

PROMETRIKA, LLC

Protalix Ltd.

QPS Austria GmbH

Rare Disease Research

Retrophin, Inc.

Sanofi Genzyme

Sigilon Therapeutics

Spark Therapeutics

Takeda Pharmaceuticals

Ultragenyx Pharmaceutical

Batten Disease Support and Research Association

Cure GMI 1 Foundation

Cure Sanfilippo Foundation

Every Life Foundation for Rare Diseases

Global Genes

Living in the Light

MLD Foundation

National Fabry Disease Foundation

National MPS Society

NORD

STAR Foundation

Alexion Pharmaceuticals

Amicus Therapeutics, Inc.

Audentes Therapeutics

Bio Insight Group

BioMarin Pharmaceutical

Carbosynth LLC

Chiesi Farmaceutici S.p.A.

Diplomat Specialty Infusion Group

Enzyvant

European Gaucher Disease Network

Fairview Specialty Pharmacy

Greenwood Genetic Center

Invitae

JCR Pharmaceuticals Co., Ltd.

Lukare Medical, LLC

Lysogene

Lysosomal & Rare Disorders Research

& Treatment Center (LDRTC)

Mayo Medical Laboratories

Pfizer, Inc.

Protalix Ltd.

QPS Austria GmbH

Retrophin, Inc.

Sangamo Therapeutics, Inc.

Sanofi Genzyme

Swedish Orphan Biovitrum AB

Takeda

Ultragenyx Pharmaceutical

Cure Sanfilippo Foundation

EveryLife Foundation for Rare Diseases

Global Genes

Jonah’s Just Begun

MLD Foundation

National MPS Society

National Organization of Rare Disorders (NORD)

Project Alive

Alexion Pharmaceuticals, Inc.

Amicus Therapeutics, Inc.

Audentes Therapeutics

Baebies

BioMarin Pharmaceutical

Carbosynth

Chiesi Farmaceutici S.p.A.

Fairview Specialty Pharmacy

GlycoAnalytics, UC San Diego

Greenwood Genetic Center

Invitae

Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)

Mayo Medical Laboratories

Pfizer, Inc.

Protalix Ltd.

QPS Austria GmbH

Rare Disease Report

Sangamo Therapeutics

Sanofi Genzyme

Shire

Ultragenyx Pharmaceutical

Batten Disease Support and Research Association

Cure Sanfilippo Foundation

EveryLife Foundation for Rare Diseases

Global Genes

Jonah’s Just Begun

LAL SOLACE

MLD Foundation

National MPS Society

National Organization of Rare Disorders (NORD)

Alexion Pharmaceuticals, Inc.

Amicus Therapeutics, Inc.

BioMarin Pharmaceutical

Carbosynth LLC

Fairview Specialty Pharmacy

GSK

Greenwood Genetic Center

Horizon Pharma

Invitae

Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)

Mayo Medical Laboratories

Pfizer, Inc.

Protalix Ltd.

Rare Disease Report

Sangamo BioSciences, Inc.

Sanofi Genzyme

Shire

ThinkGenetic Foundation

Ultragenyx Pharmaceutical

Batten Disease Support and Research Association

Fight NPC

Global Genes

Jonah’s Just Begun

LAL Solace

National Fabry Disease Foundation

National Organization of Rare Disorders

Alexion Pharmaceuticals, Inc.

American College of Medical Genetics and Genomics

Amicus Therapeutics

BioMarin Pharmaceutical Inc.

Carbosynth LLC

Emory Genetics Laboratory

Fairview Specialty Pharmacy

The Greenwood Genetic Center

Living in the Light

Lysosomal & Rare Disorders Research Center

Mayo Medical Laboratories

Orsini Healthcare

Pfizer Inc.

Protalix Biotherapeutics

Raptor Pharmaceutical Corp.

Rare Disease Report

Retrophin

Sangamo BioSciences, Inc.

Sanofi Genzyme

Shire International

Shire

Ultragenyx Pharmaceutical Inc.

Amicus Therapeutics

Alexion Pharmaceuticals, Inc.

Batten Disease Support and Research Association

BioMarin Pharmaceutical Inc.

The Fabry Support and Information Group

Genzyme, a Sanofi company

The Greenwood Genetic Center

InsideMD

The International Society for Mannosidosis and Related Diseases (ISMRD)

LAL Aware

Lukare Medical, LLC

Lysosomal & Rare Disorders Research & Treatment Center

MLD Foundation

Newborn Screening Translational Research Network (NBSTRN)

Orsini Healthcare

Pfizer Inc.

Raptor Pharmaceutical Corp.

Rare Disease Report

Shire International

Shire Pharmaceuticals

Synageva BioPharma

Ultragenyx Pharmaceutical Inc.

University of Minnesota and Fairview Pharmacy Services