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We’ve helped to redefine care in these rare and devastating diseases.

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Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)

Acute Hepatic Porphyria (AHP)

Alagille Syndrome – (ALGS)

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

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Bardet-Biedl Syndrome (BBS)

C3G and IC-MPGN

Central Precocious Puberty (CPP)

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

Congenital Adrenal Hyperplasia

Dravet Syndrome – Severe Myoclonic Epilepsy Infancy (SMEI)

Duchenne Muscular Dystrophy (DMD)

Endogenous Cushing’s Syndrome (CS)

Epidermolysis Bullosa (EB)

Familial Chylomicronemia Syndrome (FCS)

Generalized Myasthenia Gravis (gMG)

Hereditary Transthyretin Amyloidosis (hATTR)

Huntington’s Disease (HD)

Hypoparathyroidism

Hypophosphatasia (HPP)

IgA nephropathy (Berger disease)

Infantile Spasms (IS)

Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Lupus Nephritis (LN)

Mycobacterium avium complex (MAC) lung disease

Nighttime Sleep Disturbances in Smith-Magenis Syndrome (SMS)

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

Non-Cystic Fibrosis Bronchiectasis

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Prader-Willi Syndrome

Primary Biliary Cholangitis (PBC)

Primary Hyperoxaluria Type 1 (PH1)

Primary Periodic Paralysis (PPP)

Progressive Familial Intrahepatic Cholestasis (PFIC)

PSCK1, POMC, LEPR

Refractory Complex Partial Seizures

Tardive Dyskinesia (TD)

Transthyretin Amyloidosis – Cardiomyopathy (ATTR-CM)

Tuberous Sclerosis

Tumor-Induced Osteomalacia (TIO)

Type I Gaucher Disease

X-Linked Hypophosphatemia (XLH)