ZOKINVY (lonafarnib)
HUTCHINSON-GILFORD PROGERIA SYNDROME·+2
EIGER BIOPHARMA
Indication
Processing-deficient progeroid laminopathy with heterozygous LMNA mutation
Processing-deficient progeroid laminopathy with a heterozygous *LMNA* mutation is an ultra-rare, severe genetic disorder belonging to the family of premature aging syndromes. Read more about Processing-deficient progeroid laminopathy with heterozygous LMNA mutation
2 drugs 2 manufacturers
Drugs that treat it
Common Processing-deficient progeroid laminopathy with heterozygous LMNA mutation therapies.
ZOKINVY (lonafarnib)
PROGERIA SYNDROME TREATMENT AGENTS·HUTCHINSON-GILFORD PROGERIA SYNDROME·+2
SENTYNL THERAPU
Filter & compare in the drug finder →Opens the drug finder with Processing-deficient progeroid laminopathy with heterozygous LMNA mutation preselected.
News & literature
Coming soonRelated codes
Coming soonRelated conditions & guidelines
Coming soonProcessing-deficient progeroid laminopathy with a heterozygous *LMNA* mutation is an ultra-rare, severe genetic disorder belonging to the family of premature aging syndromes.
The condition is caused by a heterozygous mutation in the *LMNA* gene that impairs normal post-translational cleavage of prelamin A, leading to the toxic accumulation of permanently farnesylated, progerin-like proteins within the nuclear lamina.
This molecular defect disrupts nuclear architecture and cellular integrity, driving systemic tissue degeneration that affects pediatric and young adult patients.
Defining clinical features typically involve multiple organ systems, presenting with accelerated aging hallmarks including failure to thrive, severe lipodystrophy, alopecia, scleroderma-like skin changes, skeletal dysplasias, and early-onset, life-threatening cardiovascular disease.
- prnewswire.com
Processing-deficient progeroid laminopathy with a heterozygous *LMNA* mutation is an ultra-rare, severe genetic disorder belonging to the family of premature aging syndromes This molecular defect disrupts nuclear architecture and cellular integrity, driving systemic tissue degeneration that affects pediatric and young adult patients
- nih.gov
Processing-deficient progeroid laminopathy with a heterozygous *LMNA* mutation is an ultra-rare, severe genetic disorder belonging to the family of premature aging syndromes The condition is caused by a heterozygous mutation in the *LMNA* gene that impairs normal post-translational cleavage of prelamin A, leading to the toxic accumulation of permanently farnesylated, progerin-like proteins within the nuclear lamina This molecular defect disrupts nuclear architecture and cellular integrity, drivi...
- zokinvy.com
The condition is caused by a heterozygous mutation in the *LMNA* gene that impairs normal post-translational cleavage of prelamin A, leading to the toxic accumulation of permanently farnesylated, progerin-like proteins within the nuclear lamina
- researchgate.net
The condition is caused by a heterozygous mutation in the *LMNA* gene that impairs normal post-translational cleavage of prelamin A, leading to the toxic accumulation of permanently farnesylated, progerin-like proteins within the nuclear lamina
- aging-us.com
This molecular defect disrupts nuclear architecture and cellular integrity, driving systemic tissue degeneration that affects pediatric and young adult patients Defining clinical features typically involve multiple organ systems, presenting with accelerated aging hallmarks including failure to thrive, severe lipodystrophy, alopecia, scleroderma-like skin changes, skeletal dysplasias, and early-onset, life-threatening cardiovascular disease
- nih.gov
Defining clinical features typically involve multiple organ systems, presenting with accelerated aging hallmarks including failure to thrive, severe lipodystrophy, alopecia, scleroderma-like skin changes, skeletal dysplasias, and early-onset, life-threatening cardiovascular disease
- elsevierpure.com
Defining clinical features typically involve multiple organ systems, presenting with accelerated aging hallmarks including failure to thrive, severe lipodystrophy, alopecia, scleroderma-like skin changes, skeletal dysplasias, and early-onset, life-threatening cardiovascular disease
Personalize your experience
Follow Processing-deficient progeroid laminopathy with heterozygous LMNA mutation and get updates as they land.
Create a free account
Already a member? Sign in
Follow an indication and get alerts when its drugs, labels, or guidelines change
Save any drug on this page to your favorites, synced across devices
AI-curated peer-reviewed research, summarized for your specialty
A morning digest of indication-relevant updates, when you want it
Sign in to follow this indication
Following an indication requires a free Physicians Office Resource account.