Our Partners - PANTHERx Rare

Transthyretin Amyloidosis – Cardiomyopathy (ATTR-CM)

Rare Disorders

Specialty Areas

Cardiology

Dermatology

Epidermolysis Bullosa (EB)

Rare Disorders

Specialty Areas

Rare Disorders

Specialty Areas

Priovant Therapeutics
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Rare Disorders

Dermatomyositis

Specialty Areas

Dermatology

Central Precocious Puberty (CPP)

Rheumatology

Endocrinology

Rare Disorders

Specialty Areas

Azurity pharmaceuticals
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1-877-495-6858

Rare Disorders

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
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1-877-XERIS-37

Rare Disorders

Specialty Areas

Endogenous Cushing’s Syndrome (CS)

Rare Disorders

Specialty Areas

Hepatology

Cholestatic pruritus associated with Primary Biliary Cholangitis (PBC)

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
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1(250)744-2487

Rare Disorders

Lupus Nephritis (LN)

Specialty Areas

Mycobacterium avium complex (MAC) lung disease

Rare Disorders

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Non-Cystic Fibrosis Bronchiectasis

Rare Disorders

Specialty Areas

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

Paratek Pharmaceuticals
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617.807.6600

Rare Disorders

Specialty Areas

Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)

Rare Disorders

Specialty Areas

Priovant Therapeutics
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Rare Disorders

Dermatomyositis

Specialty Areas

Dermatology

Rheumatology

Thymidine Kinase 2 Deficiency (TK2d)

Rare Disorders

Specialty Areas

Generalized Myasthenia Gravis (gMG)

Rare Disorders

Specialty Areas

X4 Pharmaceuticals
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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

Bardet-Biedl Syndrome (BBS)

Rhythm Pharmaceuticals, Inc.
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857.264.4280

Rare Disorders

PCSK1, POMC, LEPR Deficiency

Acquired Hypothalamic Obesity (aHO)

Specialty Areas

SOLENO THERAPEUTICS a NEUROCRINE BIOSCIENCES® company
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833-SOLENO-1 (833-765-3661)

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
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1(250)744-2487

Rare Disorders

Lupus Nephritis (LN)

Specialty Areas

IgA nephropathy (Berger disease)

Calliditas Therapeutics
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844-442-0011

Rare Disorders

Specialty Areas

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Lupin Pharmaceuticals, Inc.
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866-587-4617

Rare Disorders

Specialty Areas

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Otsuka America Pharmaceutical, Inc.
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301-424-9055

Rare Disorders

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IgA nephropathy (Berger disease)

Rare Disorders

Specialty Areas

IgA nephropathy (Berger disease)

Vera Therapeutics
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(650) 555-0100

Rare Disorders

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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

SOLENO THERAPEUTICS a NEUROCRINE BIOSCIENCES® company
Visit Website
833-SOLENO-1 (833-765-3661)

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
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800.654.2299

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
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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