Manufacturer Partners

Transthyretin Amyloidosis – Cardiomyopathy (ATTR-CM)

Rare Disorders

Specialty Areas

Cardiology

Familial Chylomicronemia Syndrome (FCS)

Ionis Pharmaceuticals™
Visit Website
760-931-9200

Rare Disorders

Specialty Areas

Cardiology

Dermatology

Epidermolysis Bullosa (EB)

Rare Disorders

Specialty Areas

Rare Disorders

Specialty Areas

Rare Disorders

Specialty Areas

Azurity pharmaceuticals
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1-877-495-6858

Rare Disorders

Central Precocious Puberty (CPP)

Specialty Areas

Epidermolysis Bullosa (EB)

Rare Disorders

Specialty Areas

Rare Disorders

Specialty Areas

Neurocrine Biosciences, Inc.
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858-617‑7600

Rare Disorders

Congenital Adrenal Hyperplasia

Specialty Areas

Huntington’s Disease (HD)

Rare Disorders

Tardive Dyskinesia (TD)

Specialty Areas

Primary Periodic Paralysis (PPP)

Xeris Pharmaceuticals
Visit Website
1-877-XERIS-37

Rare Disorders

Specialty Areas

Endogenous Cushing’s Syndrome (CS)

Rare Disorders

Specialty Areas

Hepatology

Primary Biliary Cholangitis (PBC)

Rare Disorders

Specialty Areas

Hepatology

Progressive Familial Intrahepatic Cholestasis (PFIC)

Rare Disorders

Alagille Syndrome – (ALGS)

Specialty Areas

Rare Disorders

Specialty Areas

Infectious Disease & Immunology

Antineutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis (AAV)

Rare Disorders

Specialty Areas

Aurinia Pharmaceuticals
Visit Website
1(250)744-2487

Rare Disorders

Lupus Nephritis (LN)

Specialty Areas

Mycobacterium avium complex (MAC) lung disease

Rare Disorders

Specialty Areas

Non-Cystic Fibrosis Bronchiectasis

Rare Disorders

Specialty Areas

Community-Acquired Bacterial Pneumonia (CABP) and Acute Bacterial Skin and Skin Structure Infections (ABSSSI)

Paratek Pharmaceuticals
Visit Website
617.807.6600

Rare Disorders

Specialty Areas

Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)

Rare Disorders

Specialty Areas

Thymidine Kinase 2 Deficiency (TK2d)

Rare Disorders

Specialty Areas

Generalized Myasthenia Gravis (gMG)

Rare Disorders

Specialty Areas

X4 Pharmaceuticals
Visit Website
857-529-8300

Rare Disorders

WHIM Syndrome

Specialty Areas

Metabolic & Genetic Disorders

Alexion Pharmaceuticals
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475.230.2596

Rare Disorders

Hypophosphatasia (HPP)

Specialty Areas

Edenbridge Pharmaceuticals, LLC doing business as Dexcel Pharma USA
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201-292-1292

Rare Disorders

Type I Gaucher Disease

Specialty Areas

Familial Chylomicronemia Syndrome (FCS)

Ionis Pharmaceuticals™
Visit Website
760-931-9200

Rare Disorders

Specialty Areas

Cardiology

Bardet-Biedl Syndrome (BBS)

Rhythm Pharmaceuticals, Inc.
Visit Website
857.264.4280

Rare Disorders

PSCK1, POMC, LEPR

Acquired Hypothalamic Obesity (aHO)

Specialty Areas

Rare Disorders

Prader-Willi Syndrome

Specialty Areas

Thymidine Kinase 2 Deficiency (TK2d)

Rare Disorders

Specialty Areas

Generalized Myasthenia Gravis (gMG)

Rare Disorders

Specialty Areas

Nephrology

Antineutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis (AAV)

Rare Disorders

Specialty Areas

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Apellis Pharmaceutical, Inc.
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(617) 977-5700

Rare Disorders

C3G and IC-MPGN

Specialty Areas

Oncology & Hematology

Aurinia Pharmaceuticals
Visit Website
1(250)744-2487

Rare Disorders

Lupus Nephritis (LN)

Specialty Areas

IgA nephropathy (Berger disease)

Calliditas Therapeutics
Visit Website
844-442-0011

Rare Disorders

Specialty Areas

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Otsuka America Pharmaceutical, Inc.
Visit Website
301-424-9055

Rare Disorders

Specialty Areas

IgA nephropathy (Berger disease)

Rare Disorders

Specialty Areas

Neurology

Dravet Syndrome – Severe Myoclonic Epilepsy Infancy (SMEI)

Biocodex
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Rare Disorders

Specialty Areas

Harmony Biosciences
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484.539.9800

Rare Disorders

Narcolepsy

Specialty Areas

Duchenne Muscular Dystrophy (DMD)

ITF THERAPEUTICS
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Rare Disorders

Specialty Areas

Congenital Adrenal Hyperplasia

Neurocrine Biosciences, Inc.
Visit Website
858-617‑7600

Rare Disorders

Specialty Areas

Huntington’s Disease (HD)

Rare Disorders

Tardive Dyskinesia (TD)

Specialty Areas

Rare Disorders

Prader-Willi Syndrome

Specialty Areas

Huntington’s Disease (HD)

Teva Pharmaceuticals
Visit Website
1.888.838.2872

Rare Disorders

Tardive Dyskinesia (TD)

Specialty Areas

Thymidine Kinase 2 Deficiency (TK2d)

Rare Disorders

Specialty Areas

Generalized Myasthenia Gravis (gMG)

Rare Disorders

Specialty Areas

Upsher-Smith Laboratories
Visit Website
866.923.1954

Rare Disorders

Tuberous Sclerosis

Specialty Areas

Refractory Complex Partial Seizures

Rare Disorders

Infantile Spasms (IS)

Specialty Areas

Rare Disorders

Infantile Spasms (IS)

Specialty Areas

Nighttime Sleep Disturbances in Smith-Magenis Syndrome (SMS)

Vanda Pharmaceuticals
Visit Website
202.734.3400

Rare Disorders

Non-24-Hour Sleep-Wake Rhythm Disorder (N24SWD)

Specialty Areas

Primary Periodic Paralysis (PPP)

Xeris Pharmaceuticals
Visit Website
1-877-XERIS-37

Rare Disorders

Specialty Areas

Endogenous Cushing’s Syndrome (CS)

Rare Disorders

Specialty Areas

Oncology & Hematology

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Apellis Pharmaceutical, Inc.
Visit Website
(617) 977-5700

Rare Disorders

C3G and IC-MPGN

Specialty Areas

Oncology & Hematology

Pulmonary

Mycobacterium avium complex (MAC) lung disease

Rare Disorders

Specialty Areas

Non-Cystic Fibrosis Bronchiectasis

Rare Disorders

Specialty Areas