Indication
Processing-deficient progeroid laminopathy with ZMPSTE24 mutation
Processing-deficient progeroid laminopathy with ZMPSTE24 mutation refers to an ultrarare spectrum of premature aging disorders—such as mandibuloacral dysplasia type B and lethal restrictive dermopathy—caused by homozygous or compound heterozygous loss-of-function variants in the *ZMPSTE24* gene. Read more about Processing-deficient progeroid laminopathy with ZMPSTE24 mutation
2 drugs 2 manufacturers
Drugs that treat it
Common Processing-deficient progeroid laminopathy with ZMPSTE24 mutation therapies.
ZOKINVY (lonafarnib)
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Coming soonProcessing-deficient progeroid laminopathy with ZMPSTE24 mutation refers to an ultrarare spectrum of premature aging disorders—such as mandibuloacral dysplasia type B and lethal restrictive dermopathy—caused by homozygous or compound heterozygous loss-of-function variants in the *ZMPSTE24* gene.
This metalloproteinase deficiency disrupts the final proteolytic cleavage of prelamin A into mature lamin A, leading to the toxic accumulation of permanently farnesylated prelamin A within the inner nuclear membrane.
Affected pediatric patients experience widespread multiorgan degeneration characterized by severe growth failure, micrognathia, clavicular and acro-osteolysis, joint contractures, lipodystrophy, skin atrophy, and accelerated cardiovascular morbidity, driven by widespread structural nuclear envelope damage and premature cellular senescence.
- nih.gov
Processing-deficient progeroid laminopathy with ZMPSTE24 mutation refers to an ultrarare spectrum of premature aging disorders—such as mandibuloacral dysplasia type B and lethal restrictive dermopathy—caused by homozygous or compound heterozygous loss-of-function variants in the *ZMPSTE24* gene Affected pediatric patients experience widespread multiorgan degeneration characterized by severe growth failure, micrognathia, clavicular and acro-osteolysis, joint contractures, lipodystrophy, skin atro...
- zokinvy.com
Processing-deficient progeroid laminopathy with ZMPSTE24 mutation refers to an ultrarare spectrum of premature aging disorders—such as mandibuloacral dysplasia type B and lethal restrictive dermopathy—caused by homozygous or compound heterozygous loss-of-function variants in the *ZMPSTE24* gene
- nih.gov
This metalloproteinase deficiency disrupts the final proteolytic cleavage of prelamin A into mature lamin A, leading to the toxic accumulation of permanently farnesylated prelamin A within the inner nuclear membrane
- purdue.edu
This metalloproteinase deficiency disrupts the final proteolytic cleavage of prelamin A into mature lamin A, leading to the toxic accumulation of permanently farnesylated prelamin A within the inner nuclear membrane Affected pediatric patients experience widespread multiorgan degeneration characterized by severe growth failure, micrognathia, clavicular and acro-osteolysis, joint contractures, lipodystrophy, skin atrophy, and accelerated cardiovascular morbidity, driven by widespread structural n...
- nih.gov
Affected pediatric patients experience widespread multiorgan degeneration characterized by severe growth failure, micrognathia, clavicular and acro-osteolysis, joint contractures, lipodystrophy, skin atrophy, and accelerated cardiovascular morbidity, driven by widespread structural nuclear envelope damage and premature cellular senescence
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