Participating Companies

4BIO Capital Partners

Achillion Pharmaceuticals

ACMG

Aegerion Pharmaceuticals

Albireo Pharma Inc

ALD Connect

Alea Diagnostics

Alexion Pharmaceuticals Inc

Alliance for Regenerative Medicine

Altius Strategy Consulting

Amyloidosis Research Consortium

Anima Biotech Inc

APR Applied Pharma Research S A

Asahi Kasei Pharma

Asklepios BioPharmaceuticals

Athersys Inc

AveXis

Berg Health

Bexion

Biogen

Biomatrix

bluebird bio

Blueprint Medicines Corporation

Boston Childrens Hospital

Brazilian Pulmonary Hypertension As

Brazilian Pulmonary Hypertension As

CCHS Famliy Network

Chiesi

Children's Hospital Association

Cincinnati Childrens Hospital

Clinical Network Services Pty. Ltd.

Continga Pharamceuticals

Corbus Pharmaceuticals

CSI Specialty Group

Cure CMT4J

Cure Sanfilippo Foundation

CureCADASIL

CureDuchenne

CureDuchenne

Cures Within Reach

Cydan II Inc

Cystic Fibrosis Research, Inc. (CFR

Debra of America

Dup15q Alliance

Durbin PLC

DyDo Pharma Inc

DyDo Pharma INC.

Enclear Therapies

Everylife Foundation for Rare Disea

Familial Dysautonomia Foundation

Fed. Colombiana de Enfermedades Rar

Food & Drug Administration

Foundation for USP7 Related Disease

Geisinger

Girls Chronically Rock LLC

Global Genes

Greater Boston Sickle Cell Disease

Healx Limited

Horizon Pharma

Hyundai Pharm

Inceptua Ltd

Intercept Pharmaceuticals

International Rett Syndrome Foundat

Ipsen

IPSEN BioSciences

Jaguar Health, Inc.

JP Morgan Chase

JPA Health Communications

LEO Pharma Inc

Li Fraumeni Syndrome Association

Little Hercules Foundation

Marinus Pharmaceuticals Inc

Massachusetts Biotechnology Council

Massachusetts General Hospital

Medical Marketing Economics LLC

Mesothelioma Applied Research Found

MGH Harvard Medical School

MIT Sloan School of Mgmt

ML4 Foundation

MPN Research Foundation

MTM-CNM Family Connection, Inc.

Myasthenia Gravis Association

Myositis Association

National Hemophilia Foundation

NewBridge Pharmaceuticals

Nobelpharma Co Ltd

Notable Labs

Org for Rare Diseases India, USA

Parent Project Muscular Dystrophy (PPMD)

PathAI

Perlara PBC

Pfenex Inc

Pfizer

Pharming Healthcare

Phoenix Tissue Repair

PHS Solutions

Protean LLC

PTEN Hamartoma Tumor Syndrome Found

RareLife Solutions

Raremark

Recordati Rare Diseases Inc

Recursion Pharmaceuticals

Retrophin

Rho World

Rhythm Pharmaceuticals

SCRI

Sherry Fox

SLC6A1 Connect

Sobi Inc

STATKING Clinical Services

Swoop

SYNGAP Education & Research Foundat

Syros Pharmaceuticals

Target Health Inc

The Jackson Laboratory

The Neuromuscular Disease Foundatio

The Oley Foundation

TSF

Tufts University School of Medicine

Two Labs Pharma Services

United MSD Foundation

Vistrisa Therapeutics

VOZ Advisors

WAVE Life Sciences

Weill Cornell Medical College

Xontogeny