Oral small moleculetherapyfor lysosomal storage diseases
In addition, prenatal diagnosis of lysosomal storage disease
can be made by analysis of glycolipids and oligosaccharides in the amniotic fluid using a method called "electrospray ion tandem mass spectrometry" (14).
Insights into the diagnosis and treatment of lysosomal storage diseases
Working with animal models of a group of fatal neurological disorders called lysosomal storage diseases
, the researchers found that these diseases cause unique and disease-specific alterations to the blood vessels of the blood brain barrier.
Malfunctions in the autophagy process have been reported in other lysosomal storage diseases
, in addition to diseases such as Parkinsons and Alzheimers diseases, Zheng said.
MONTREAL -- Angiochem announced today a global collaboration with GlaxoSmithKline (GSK) to discover, develop and commercialize treatments for lysosomal storage diseases
M2 PHARMA-February 8, 2017-US FDA Grants de Novo Clearance for Baebies' Lysosomal Storage Disease
Newborn Screening Platform
This research collaboration will be the basis for a development program for therapies for multiple such rare diseases, including Lysosomal Storage Diseases
The company may also use the funds to move the programmes of ulcerative colitis, repair of brain and spinal injury, the treatment of muscular dystrophies, and the treatment of lysosomal storage diseases
, into active phases with the intention of identifying at least three candidate drugs to be made clinic-ready within two years.
We chose lysosomal storage diseases
because of their unmet need and existing opportunities for developing and marketing more ERTs.
These include lysosomal storage diseases
and other progressive, serious and life-threatening genetic conditions.
NPC is reportedly one of at least 42 rare, fatal diseases which are classed as lysosomal storage diseases