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Caring for Calcium-Related Rare Diseases

Caring for Calcium-Related Rare Diseases

A Start Up founded in December 2016 and committed to the discovery and development of orphan drugs for patients affected by calcium-related genetic diseases. ChemICare’s mission is the improvement of the quality of life of patients affected by Tubular Aggregate Myopathy, York Platelet Syndrome and Stormorken Syndrome by the development of a therapy that restores the abnormal intracellular calcium level, a common feature of these three rare diseases.

 

 

 

 

ChemICare’s efforts have resulted in the generation of a number of unique and potent inhibitors of the Store Operated Calcium Entry. These compounds have demonstrated to restore the intracellular calcium levels to physiological levels, to be effective in biopsies of patients affected by Tubular Aggregate Myopathy and are currently being evaluated in transgenic mouse models. We believe these molecules could represent a therapeutic treatment for people affected by rare pathologies related to gain-of-function mutations of STIM1 and Orai1.

DISEASES

ChemICare targets a cluster of rare diseases currently without therapy: Tubular Aggregate Myopathy (TAM), characterized by painful contractures and progressive skeletal muscle degeneration, York platelet Syndrome (YPS), where the main hallmark is the abnormal bleeding, and Stormorken Syndrome (STRMK), a combination of the previous two pathologies. These three diseases are caused by gain-of-function mutations in Orai1 and STIM1, which cause an abnormal increase in intracellular calcium levels.  MORE

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TARGET

ChemICare’s research is focused on a cellular event called Store Operated Calcium Entry, where the depletion of calcium from the endoplasmic reticulum triggers calcium influx across the plasma membrane. Two main proteins are involved: STIM, the calcium sensor in the endoplasmic reticulum, and Orai, the ion channel selective for calcium ion.  MORE                                                                                                                                                                                                                                                                                                                     .

Different rare diseases

Millions people affected worldwide

%

Affect children

%

Has a drug treatment

 

 

 

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ChemICare srl  – P:IVA 02302680034

Via Amico Canobio 4/6, Novara

info@chemicare.it

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