Thelansis

  Adrenomyeloneuropathy (AMN) is an inherited condition that affects the spinal cord. It is a form of X-linked adrenoleukodystrophy (X-ALD), characterized by spastic paraparesis and often associated with peripheral adrenal insufficiency in males. Etiology- AMN is due to mutations of ABCD1 (Xq28) encoding ALDP, a peroxisomal transmembrane protein involved in the transport of very long-chain fatty acid-CoA esters (VLCFA) into the peroxisome. Oxidative stress and damage to proteins and lipids due to VLCFA accumulation in glial cells (oligodendrocytes, Schwann cells) may impair their capacity to sustain axonal integrity in the spinal cord and peripheral nerves. Epidemiology- X-ALD estimated birth incidence is 1/20,000. Adrenomyeloneuropathy (AMN) manifests in more than 60% of female patients in adulthood and nearly all male patients who reach adulthood. The competitive landscape of Adrenomyeloneuropathy (AMN) includes country-specific approved and pipeline therapies. Any asse...