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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)
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EXPLORE
Hereditary Transthyretin Amyloidosis (hATTR)
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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)
Tuberous Sclerosis
Tumor-Induced Osteomalacia (TIO)
Type I Gaucher Disease
WHIM Syndrome
Wilson Disease
X-Linked Hypophosphatemia (XLH)
