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Rare Disorders
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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)
Central Precocious Puberty (CPP)
Dravet Syndrome – Severe Myoclonic Epilepsy Infancy (SMEI)
Duchenne Muscular Dystrophy (DMD)
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)
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)
Progressive Familial Intrahepatic Cholestasis (PFIC)
PSCK1, POMC, LEPR
Refractory Complex Partial Seizures
Tardive Dyskinesia (TD)
Tuberous Sclerosis
Tumor-Induced Osteomalacia (TIO)
Type I Gaucher Disease
WHIM Syndrome
Wilson Disease
X-Linked Hypophosphatemia (XLH)
