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Rare Disorders
We've helped to redefine care in these rare and devastating diseases.
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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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