ION717 clinical trial completes patient enrolment
Autor: Eric Minikel (traducido por Joaquín Castilla) Ionis Pharmaceuticals ha anunciado (ver la carta original en inglés) esta mañana que ha completado la inscripción de PrProfile, su ensayo clínico de Fase 1/2a del ION717, un fármaco ASO diseñado para reducir los niveles de PrP en las enfermedades priónicas. El estudio ahora aparece como «Activo, sin […]
Scientists discover early brain alterations in people at risk of developing hereditary Creutzfeldt-Jakob disease
Author: Cristina Sampedro Prion diseases can occur spontaneously or genetically, the latter being caused by a mutation in the prion protein (PrP), which are rare but devastating neurodegenerative disorders that affect both humans and animals. The main types of genetic prion diseases are genetic Creutzfeldt-Jakob disease (gCJD), genetic Creutzfeldt-Jakob disease (gCJD), genetic Creutzfeldt-Jakob disease (gCJD), genetic [...]
Amyotrophic Lateral Sclerosis joins the group of diseases known as prion-like diseases.
Author: Jorge Moreno Charco Prion diseases, or Transmissible Spongiform Encephalopathies (TSE), could be defined as a group of neurodegenerative diseases related to the misfolding and propagation of the endogenous prion protein through the central nervous system. This misfolded protein, in addition to spreading uncontrollably through the patient's brain, is highly neurotoxic, which can lead to a [...]
Research priorities on the role of α-synuclein in the pathogenesis of Parkinson's Disease
Authors: Nerea Larrañaga and Paula Marco Scientists from the International Parkinson and Movement Disorders Society (MDS) have published a paper highlighting the main research priorities for α-synuclein (αSyn) protein research in Parkinson's disease. This review, led by an international team of experts, focuses on addressing key aspects of α-synuclein [...]
Global Update on Prion Diseases: What does the latest Epidemiological Atlas tell us?
Authors: Paula Marco and Nerea Larrañaga Human prion diseases (such as Creutzfeldt-Jakob disease (CJD), fatal familial insomnia (FFI) and Gerstmann-Sträussler-Scheinker syndrome (GSS), among others) are a group of rapidly progressive, fatal and potentially transmissible neurodegenerative disorders caused by the misfolding of the physiological protein PrPC, which results in the misfolding of the protein PrPC.
Expansion and reactivation of Ionis PrProfile (ION717) clinical trial for people with symptomatic prion disease
Author: Joaquín Castilla Ionis Pharmaceuticals has just announced that the recruitment pause for its PrProfile (ION717) study for people with symptomatic prion disease has been lifted. Important information to note: Resumption of recruitment: After a planned pause, recruitment for the PrProfile clinical trial has resumed as of [...]
New international study looks for correlations between genetic variations and sporadic CJD
Author: Josu Galarza Scientists from around the world have collaborated on a genetic study that seeks to identify small changes in the genome that may be related to the clinical development and age of onset of sporadic Creutzfeldt-Jakob disease (sCJD). Prion diseases are classified as rare diseases due to their low incidence [...].
New strategies to understand and combat sporadic Creutzfeldt-Jakob disease through integration of genetic data
Autor: Nuno Anjo La enfermedad de Creutzfeldt-Jakob esporádica (sCJD) es una enfermedad neurodegenerativa rara, fatal y de rápida progresión, para la cual aún no existen tratamientos efectivos. Se caracteriza por el malplegamiento de la proteína priónica normal (PrPC) en una forma anómala llamada PrPSc o Prion, que se acumula en el cerebro, causando daño neuronal […]
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