Rare Disorders

Acute Hepatic Porphyria (AHP)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Central Precocious Puberty (CPP)

Gastrointestinal Stromal Tumors (GIST)

Gastrointestional Stromal Tumors (GIST)

Hypophosphatasia (HPP)

Infantile Spasms (IS)

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

Mycobacterium avium complex (MAC) lung disease

Narcolepsy

Neuromyelitis Optica Spectrum Disorder (NMOSD)

Nighttime Sleep Disturbances in Smith-Magenis Syndrome (SMS)

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

Non-Small Cell Lung Cancer (NSCLC)

Primary Periodic Paralysis (PPP)

RET+ve Thyroid Cancer

Tardive Dyskinesia (TD)

Tumor Induced Osteomalacia (TIO)

X-Linked Hypophosphatemia (XLH)

Soon to Be Announced

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