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Arimoclomol
Arimoclomol, sold under the brand name Miplyffa, is a medication used for the treatment of Niemann–Pick disease type C. It is taken by mouth.
The most common side effects include upper respiratory tract infection, diarrhea, and decreased weight.
Arimoclomol was approved for medical use in the United States in September 2024. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.
Arimoclomol, in combination with miglustat, is indicated for the treatment of neurological symptoms associated with Niemann-Pick disease, type C (NPC) in adults and children two years of age and older.
The most common side effects of arimoclomol include upper respiratory tract infection, diarrhea, and decreased weight.
Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones.[medical citation needed] Since damaged proteins, called aggregates, are thought to play a role in many diseases, CytRx believes that arimoclomol could treat a broad range of diseases.[medical citation needed]
Arimoclomol activates the heat shock response. It is believed to act at Hsp70.
Arimoclomol was discovered by Hungarian researchers, as a drug candidate to treat insulin resistance and diabetic complications such as retinopathy, neuropathy and nephropathy. Later, the compound, along with other small molecules, was screened for further development by Hungarian firm Biorex, which was sold to CytRx Corporation, who developed it toward a different direction from 2003.
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Arimoclomol
Arimoclomol, sold under the brand name Miplyffa, is a medication used for the treatment of Niemann–Pick disease type C. It is taken by mouth.
The most common side effects include upper respiratory tract infection, diarrhea, and decreased weight.
Arimoclomol was approved for medical use in the United States in September 2024. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.
Arimoclomol, in combination with miglustat, is indicated for the treatment of neurological symptoms associated with Niemann-Pick disease, type C (NPC) in adults and children two years of age and older.
The most common side effects of arimoclomol include upper respiratory tract infection, diarrhea, and decreased weight.
Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones.[medical citation needed] Since damaged proteins, called aggregates, are thought to play a role in many diseases, CytRx believes that arimoclomol could treat a broad range of diseases.[medical citation needed]
Arimoclomol activates the heat shock response. It is believed to act at Hsp70.
Arimoclomol was discovered by Hungarian researchers, as a drug candidate to treat insulin resistance and diabetic complications such as retinopathy, neuropathy and nephropathy. Later, the compound, along with other small molecules, was screened for further development by Hungarian firm Biorex, which was sold to CytRx Corporation, who developed it toward a different direction from 2003.