Mucopolysaccharidosis
type III (MPS III), also known as Sanfilippo syndrome, is a progressive disorder that
primarily affects brain and spinal cord (central nervous system). People with
MPS III generally do not display features at birth, but they begin to show
signs and symptoms of this disorder during early childhood. Affected children often
initially have delayed speech and behaviour problems. In later stages of this
disorder, people with MPS III may develop seizures and movement disorders.
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Detailed Report Summary: https://www.pharmaproff.com/report/mps-iiitherapeutics-pipeline-analysis
Patients
with MPS III are also known to have short stature, joint stiffness, or mild
dysostosis multiplex. Affected individuals often experience chronic diarrhoea
and recurrent upper respiratory and ear infections. People with MPS III may
also have hearing loss and vision problems. MPS III is divided into four types;
IIIA, IIIB, IIIC, and IIID, which are distinguished by their genetic cause.
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The drug
candidates in MPS III pipeline include, but are not limited to, EGT-101, MPS
IIIA (Sanfilippo A) Program and LYS-SAF302. Some companies having drugs in the
MPS III pipeline are Esteve Pharmaceuticals S.A., Orchard Therapeutics Limited,
Lysogene S.A. among others.
The report
provides a comprehensive understanding of the pipeline activities covering all
drug candidates under various stages of development, with the detailed analysis
of pipeline and clinical trials.
Pipeline
analysis of drugs by phases includes product description and development
activities including information about clinical results, designations,
collaborations, licensing, grants, technology, and others.
