Disease-modifying therapy for proteinopathies: Can the exception become the rule?
Prog Mol Biol Transl Sci
; 168: 277-287, 2019.
Article
em En
| MEDLINE
| ID: mdl-31699321
ABSTRACT
Disease-modifying therapies for proteinopathies are urgently needed yet clinical trials for the major neurodegenerative diseases, Alzheimer's and Parkinson's, have been failing at an alarming rate leaving patients and caregivers scrambling for any sign of hope. At the same time, for one family of proteinopathies, the rare TTR amyloidoses, disease-modifying therapy has existed for almost 3 decades and two new types of disease-modifying therapy have become available more recently. In this chapter, I discuss those therapies, examine to what extent they can be generalized for other diseases, and consider what we may learn from their relative success.
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Texto completo:
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Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Doenças Neurodegenerativas
/
Deficiências na Proteostase
Limite:
Animals
/
Humans
Idioma:
En
Revista:
Prog Mol Biol Transl Sci
Ano de publicação:
2019
Tipo de documento:
Article