Rare Diseases Symptoms Automatic Extraction

Therapeutic modulation of Notch signalling--are we there yet?

[cadasil]

The Notch signalling pathway is evolutionarily conserved and is crucial for the development and homeostasis of most tissues. Deregulated Notch signalling leads to various diseases, such as T cell leukaemia, Alagille syndrome and a stroke and dementia syndrome known as CADASIL, and so strategies to therapeutically modulate Notch signalling are of interest. Clinical trials of Notch pathway inhibitors in patients with solid tumours have been reported, and several approaches are under preclinical evaluation. In this Review, we focus on aspects of the pathway that are amenable to therapeutic intervention, diseases that could be targeted and the various Notch pathway modulation strategies that are currently being explored.

Diseases presenting "stroke" symptom

  • acute rheumatic fever
  • adrenomyeloneuropathy
  • alexander disease
  • alpha-thalassemia
  • cadasil
  • cohen syndrome
  • dedifferentiated liposarcoma
  • fabry disease
  • heparin-induced thrombocytopenia
  • hereditary cerebral hemorrhage with amyloidosis
  • homocystinuria without methylmalonic aciduria
  • hydrocephalus with stenosis of the aqueduct of sylvius
  • kallmann syndrome
  • locked-in syndrome
  • malignant atrophic papulosis
  • neuralgic amyotrophy
  • sneddon syndrome
  • thoracic outlet syndrome
  • werner syndrome
  • zellweger syndrome

This symptom has already been validated