Heat shock proteins, such as HSP40, HSP60, HSP70 and HSP90, are known as molecular chaperones because they play a role in helping proteins function properly when cells are under stress. The heat shock response protects cells from the accumulation of misfolded proteins, aggregated proteins, and dysfunctional lysosomes (the cell’s recycling system), which could otherwise lead to toxicity and disease.
Our work is focused on making use of the cell-protective properties of HSP70 to create new therapies for rare neurodegenerative diseases.
We have clinical trial programs underway to explore the potential of our lead investigational treatment, arimoclomol, on two types of disease areas lysosomal storage diseases (LSDs) – specifically Niemann-Pick disease type C (NPC) and Gaucher disease – and neuromuscular diseases – specifically Amyotrophic Lateral Sclerosis (ALS) and Inclusion Body Myositis (IBM).
Orphazyme A/S Company announcement No. 12/2021 Inside information Company Registration No. 32266355 Copenhagen – March 29, 2021 – Orphazyme A/S [ORPHA.CO (DK); ORPH (US)], a late-stage biopharmaceutical company
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