The company has a development portfolio of five product candidates, with the option to license seven more, with lead programs in GM1 gangliosidosis, frontotemporal dementia (FTD) and Krabbe disease
, all of which are planned to be in the clinic in 2020.
Expectant families may be eligible for this cost free programme if a family member has been diagnosed with certain cancers, such as leukemia, or blood, immune, and metabolic disorders, such as sickle cell anemia, DiGeorge syndrome, and Krabbe disease
, which may require a stem cell transplant.
Colin Brown, from Upper Largo, Fife, is coming to terms with losing his daughter Eilish, who was diagnosed with Krabbe Disease
and died aged just nine months in 2014.
5, 2005, Hunter Kelly, the son of former Buffalo Bills quarterback Jim Kelly, lost the battle against globoid-cell Leukodystrophy, or Krabbe Disease
The MSC2 phenotype, with its enhanced anti-inflammatory function, has therapeutic potential against demyelinating disorders (e.g., optic neuritis, multiple sclerosis, and Krabbe disease
), diabetic neuropathy, and inflammatory disorders (e.g., rheumatoid arthritis and Crohn's disease).
Less well known are the fatal early-onset neurodegenerative diseases such as Krabbe disease
that primarily affect young children.
may mimic GAN with early cerebellar involvement and is more common.
She suffered from a rare genetic condition called Krabbe Disease
, which affects the nervous system.
(globoid cell leukodystrophy) is an often-fatal lysosomal storage disease caused by the deficiency of the enzyme galactocerebrosidase (GALC; (8) EC 126.96.36.199), resulting in the loss of the myelin sheath in the central nervous system (1, 2).
Without it, the boys - who suffer from Krabbe disease
which destroys young bodies - would most likely be dead.
(9) Amongst those involving predominantly white matter can be further categorised as predominant involvement of periventricular white matter as in Metachromatic leukodystrophy, Classic X-linked adrenoleukodystrophy, Globoid cell leukodystrophy (Krabbe disease
), vanishing white matter disease and those with predominant subcortical white matter involvement as Van Der Knaap disease.
The product is intended for the treatment of Type 1 - Early Infantile Krabbe disease
, a rare genetic degenerative disorder for which there is no cure and is generally fatal before two years of age.