Bae et al., "Hutchinson-Gilford progeria syndrome
with G608G LMNA mutation," Journal of Korean Medical Science, vol.
Rapamycin activates autophagy in Hutchinson-Gilford progeria syndrome
: Implications for normal aging and age-dependent neurodegenerative disorders.
Progeria, also known as Hutchinson-Gilford Progeria Syndrome
(HGPS), is a rare and rapidly fatal genetic condition of accelerated ageing in children caused by a point mutation in the lamin A gene yielding the farnesylated aberrant protein, progerin.
Having a new tool to study aging could help us make new discoveries, for example to treat genetic predispositions where aging starts earlier, such as Hutchinson-Gilford progeria syndrome
," she says.
Hutchinson-Gilford Progeria Syndrome
is a genetic disease characterised by the dramatic, rapid appearance of ageing, starting from childhood.
It turned out that Sam has Hutchinson-Gilford progeria syndrome
, an extremely rare genetic disease that affects about one in 4 million to 8 million children.
researchers, who include Francis Collins, one of the world's most eminent scientists, hope the finding will lead to new ways of treating Hutchinson-Gilford Progeria Syndrome
, a rare genetic condition in which babies rapidly grow old and frail, before dying of 'old age' at around 12.
M2 PHARMA-December 4, 2018-Eiger submits lonafarnib IND with US FDA for the treatment of Hutchinson-Gilford Progeria Syndrome
and progeroid laminopathies
The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation (RPD) to United States-based Eiger BioPharmaceuticals' lonafarnib, intended to treat both Hutchinson-Gilford Progeria Syndrome
(HGPS or progeria) and progeroid laminopathies, it was reported on Tuesday.
Eiger BioPharmaceuticals (EIGR) has expanded its licensing agreement with Merck (MRK) to include rights to develop the investigational farnesyltransferase inhibitor lonafarnib for the treatment of Hutchinson-Gilford Progeria Syndrome
, a rare and fatal genetic condition characterized by accelerated aging in children.