Hereditary ATTR amyloidosis (hATTR) is a progressive condition caused by a mutation or error in the TTR (transthyretin) gene...
X-Linked Myotubular Myopathy (XLMTM), is a rare and life-threatening neuromuscular disease that is characterized by...
In the midst of conducting a clinical trial, Biogen had to confront that very few target enrollees were actually trial-eligible…
Nontuberculous Mycobacterial Lung Diseases (NTM) is a progressive and chronic condition that causes severe...
Warm autoimmune hemolytic anemia (wAIHA) is an autoimmune disorder characterized by the premature destruction...
WHIM Syndrome is a rare immuno-deficiency caused by gain-of-functions mutations in the C-X-C chemokine...
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