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Advances in orphan drug development for alpha-1 antitrypsin deficiency: a 2025 update from the FDA and EMA. [PDF]

open access: yesTher Adv Respir Dis
Höger P   +8 more
europepmc   +1 more source
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Farnesyltransferase inhibitors versus Ras inhibitors

Current Opinion in Chemical Biology, 1997
Over the past few years, the idea that farnesyl-protein transferase (FPTase) inhibitors might be effective antiproliferative/antitumor agents has been realized in studies of cultured cells and in rodent models of cancer. Most of the studies with FPTase inhibitors have focused on inhibiting the growth of ras-transformed cells in vitro or the growth of ...
J B, Gibbs   +6 more
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Sirtuin inhibitors

Expert Opinion on Therapeutic Patents, 2009
The sirtuin family of deacetylase enzymes comprises seven proteins (SIRT1-7) that are dependent on NAD(+) for their activity. Three proteins are located in the nucleus, three in the mitochondria and only one is predominantly cytoplasmic. Caloric restriction and oxidative stress generally up-regulate their expression. SIRT1, the orthologue of yeast Sir2,
Francisco J, Alcaín, José M, Villalba
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Renin Inhibitors

Pharmaceutical Research, 1987
Since the early 1980s, an intensive effort has been focused on the development of orally effective and long-acting inhibitors of renin. During this time, in vitro potency has increased greatly, with several transition-state inhibitor designs yielding inhibitors with subnanomolar IC50 values.
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