Eiger is preparing an NDA and MAA for lonafarnib to treat Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and Progeroid Laminopathies
David Cory, President and CEO of Eiger, said, 'Eiger's pipeline is now late-stage with planned global regulatory activities including an NDA and MAA for Progeria and Progeroid Laminopathies
, enrollment of a global Phase 3 study for Lonafarnib in HDV, and End of Phase 2 meetings for Peginterferon Lambda in HDV and Avexitide in Post-Bariatric Hypoglycemia.
Clinigen Group has partnered with Eiger BioPharmaceuticals to launch a worldwide lonafarnib Managed Access Program for patients with Progeria and Progeroid Laminopathies
. Progeria, also known as Hutchinson-Gilford Progeria Syndrome, is a rare and fatal genetic condition of accelerated aging in children that researchers believe is caused by a genetic mutation that results in an overabundance of the farnesylated aberrant protein named progerin.
Global Banking News-December 20, 2018-Eiger awarded US FDA's breakthrough therapy designation for lonafarnib for the treatment of Progeria and progeroid laminopathies
These proteins play an important role in many cellular processes, such as in providing a nuclear scaffold for protein complexes, maintaining nuclear structure, regulating gene expression, and playing roles in signaling pathways.,,, Mutations in the LMNA gene cause a series of rare diseases known as laminopathies
, which involve skeletal muscles, cardiac muscles, peripheral nerves and fat tissue., Skeletal muscle disorders caused by mutations in the LMNA gene can manifest as diverse phenotypes, such as LMNA -associated congenital muscular dystrophy (L-CMD), Emery-Dreifuss muscular dystrophy (EDMD), and limb-girdle muscular dystrophy type 1B (LGMD1B).
Rewritten, reorganized, and updated, this edition has 19 new plates and includes an overview of human evolution, aging, the CRISPR (Cluster Regularly Interspaced Short Palindromic Repeats)-Cas principle, genetic signaling pathways, genomic disorders and genome-wide association studies, cancer genomes, laminopathies
, chromatin disorders, cohesinopathies, and other emerging topics.
Mutalif et al., "Accumulation of the inner nuclear envelope protein Sun1 is pathogenic in progeric and dystrophic laminopathies
," Cell, vol.
Shackleton, "A novel interaction between lamin a and SREBP1: implications for partial lipodystrophy and other laminopathies
," Human Molecular Genetics, vol.
These proteins are key players in cellular senescence, as illustrated by their genetic depletion, which causes early onset of senescence, comprising a group of rare genetic diseases collectively referred to as laminopathies
. In natural senescence, LmnA seems to remain constant over time whereas LmnC decreases significantly [17,18], although this could be cell-type dependent, since other studies report a concomitant decrease of both isoforms .
Such cells with abnormal nucleus are prone to develop several diseases which are collectively referred to as laminopathies
[27,28] such as Emery-Dreifuss muscular dystrophy [29,30], Dunnigantype familial partial lipodystrophy [31,32], and mandibuloacral dysplasia [33,34].
Mutations in Lmna are responsible for more than ten different disorders, commonly referred to as "laminopathies
A group of human diseases called laminopathies
, which include premature aging, are caused by defects in proteins called lamins.