ONPATTRO (patisiran sodium, lipid complex)
AMYLOIDOSIS AGENTS - TRANSTHYRETIN (TTR) SUPPRESSION · TRANSTHYRETIN FAMILIAL AMYLOID POLYNEUROPATHY
ALNYLAM PHARMAC
Drug class
Amyloidosis Agents - Transthyretin (TTR) Suppression
Amyloidosis agents that suppress transthyretin comprise a therapeutic category of targeted genetic therapies, specifically small interfering RNAs and antisense oligonucleotides, designed to reduce the hepatic synthesis of the transthyretin protein. Read more about Amyloidosis Agents - Transthyretin (TTR) Suppression
4 drugs in this class 3 manufacturers
Drugs in Amyloidosis Agents - Transthyretin (TTR) Suppression
TEGSEDI (inotersen sodium)
AMYLOIDOSIS AGENTS - TRANSTHYRETIN (TTR) SUPPRESSION · TRANSTHYRETIN FAMILIAL AMYLOID POLYNEUROPATHY
AKCEA THERAPEUT
AMVUTTRA (vutrisiran sodium)
AMYLOIDOSIS AGENTS - TRANSTHYRETIN (TTR) SUPPRESSION · TRANSTHYRETIN FAMILIAL AMYLOID POLYNEUROPATHY ·+1
ALNYLAM PHARMAC
WAINUA (eplontersen sodium)
AMYLOIDOSIS AGENTS - TRANSTHYRETIN (TTR) SUPPRESSION · TRANSTHYRETIN FAMILIAL AMYLOID POLYNEUROPATHY
AstraZeneca Pharmaceuticals
Filter & compare in the drug finder →
Opens the drug finder with Amyloidosis Agents - Transthyretin (TTR) Suppression preselected. Add indication or manufacturer filters there.
Amyloidosis agents that suppress transthyretin comprise a therapeutic category of targeted genetic therapies, specifically small interfering RNAs and antisense oligonucleotides, designed to reduce the hepatic synthesis of the transthyretin protein.
By binding to transthyretin messenger RNA and promoting its degradation, these agents substantially lower circulating levels of both mutant and wild-type protein, thereby preventing the formation and systemic deposition of pathogenic amyloid fibrils.
Clinically, this drug class is indicated for managing transthyretin-mediated amyloidosis, predominantly treating hereditary transthyretin amyloid polyneuropathy and transthyretin amyloid cardiomyopathy.
-
nih.gov
Amyloidosis agents that suppress transthyretin comprise a therapeutic category of targeted genetic therapies, specifically small interfering RNAs and antisense oligonucleotides, designed to reduce the hepatic synthesis of the transthyretin protein By binding to transthyretin messenger RNA and promoting its degradation, these agents substantially lower circulating levels of both mutant and wild-type protein, thereby preventing the formation and systemic deposition of pathogenic amyloid fibrils Cl...
-
arci.org
Amyloidosis agents that suppress transthyretin comprise a therapeutic category of targeted genetic therapies, specifically small interfering RNAs and antisense oligonucleotides, designed to reduce the hepatic synthesis of the transthyretin protein By binding to transthyretin messenger RNA and promoting its degradation, these agents substantially lower circulating levels of both mutant and wild-type protein, thereby preventing the formation and systemic deposition of pathogenic amyloid fibrils
-
fda.gov
Clinically, this drug class is indicated for managing transthyretin-mediated amyloidosis, predominantly treating hereditary transthyretin amyloid polyneuropathy and transthyretin amyloid cardiomyopathy
Sign in to follow this class
Following a drug class requires a free Physicians Office Resource account.