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Medications
Rare Disorders
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Patients
Manufacturers
Our Partners
Prescribers
Payers
About Us
Resources
Careers
Contact Us
Medications
Rare Disorders
Specialty Areas
Medications
We’ve helped to redefine care in these rare and devastating diseases.
Medications
Rare Disorders
Our Partners
Specialty Areas
Medications
Rare Disorders
Our Partners
Specialty Areas
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Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)
Acute Hepatic Porphyria (AHP)
Alagille Syndrome – (ALGS)
Amyotrophic Lateral Sclerosis (ALS)
Antineutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis (AAV)
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Bardet-Biedl Syndrome (BBS)
Central Precocious Puberty (CPP)
Dravet Syndrome – Severe Myoclonic Epilepsy Infancy (SMEI)
Duchenne Muscular Dystrophy DM
Endogenous Cushing’s Syndrome (CS)
Epidermolysis Bullosa (EB)
Gastrointestional Stromal Tumors (GIST)
Generalized Myasthenia Gravis (gMG)
EXPLORE
EXPLORE
Hereditary Transthyretin Amyloidosis (hATTR)
EXPLORE
EXPLORE
Huntington’s Disease (HD)
Hypophosphatasia (HPP)
IgA nephropathy (Berger disease)
Infantile Spasms (IS)
Leptin Receptor (LEPR) Deficiency Obesity
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Lupus Nephritis (LN)
Mycobacterium avium complex (MAC) lung disease
Narcolepsy
Nighttime Sleep Disturbances in Smith-Magenis Syndrome (SMS)
Non-24-Hour Sleep-Wake Rhythm Disorder (N24SWD)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Primary Biliary Cholangitis (PBC)
Primary Hyperoxaluria Type 1 (PH1)
Primary Periodic Paralysis (PPP)
Pro-Opiomelanocortin (POMC)
Progressive Familial Intrahepatic Cholestasis (PFIC)
Proprotein Convertase Subtilisin/Kexin type 1 (PCSK1)
Refractory Complex Partial Seizures
Tardive Dyskinesia (TD)
Transthyretin Amyloidosis – Cardiomyopathy (ATTR-CM)
Tuberous Sclerosis
Tumor-Induced Osteomalacia (TIO)
Type I Gaucher Disease
WHIM Syndrome
Wilson Disease
X-Linked Hypophosphatemia (XLH)
