lysosomal storage disorder Companies
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based in Oxford, UNITED KINGDOM
Evox Therapeutics is a privately held, Oxford-based biotechnology company focused on improving the natural delivery capabilities of exosomes, and developing an entirely new class of therapeutics. Backed by leading venture capital groups and ...
Evox - Model Type C - Lysosomal Storage Disorder
Niemann-Pick C disease (NPC) is a lysosomal storage disorder which affects the brain and multiple other ...
REQUEST QUOTEEvox Therapeutics is a privately held, Oxford-based biotechnology company focused on improving the natural delivery capabilities of exosomes, and developing an entirely new class of therapeutics. Backed by leading venture capital groups and ...
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based in Bethesda, MARYLAND (USA)
Gain Therapeutics is redefining drug discovery with its SEE-Txâ„¢ target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for ...
Gain - Protein Misfolding Technology
Protein misfolding is the root of many diseases, including lysosomal storage disorders and neurodegenerative ...
REQUEST QUOTEGain Therapeutics is redefining drug discovery with its SEE-Txâ„¢ target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for ...
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based in Rostock, GERMANY
Since 2006, CENTOGENE has helped physicians diagnose thousands of rare disease patients around the world. A quick and accurate diagnosis can end an odyssey of medical tests and visits to multiple medical specialists. It also helps physicians better ...
Genetic Testing Services
Genetic testing is a type of medical test that identifies changes in genes, inherited from our parents, which we then typically pass on to our children. “Mistakes” in our genes (called “pathogenic variants”) can manifest the ...
REQUEST QUOTESince 2006, CENTOGENE has helped physicians diagnose thousands of rare disease patients around the world. A quick and accurate diagnosis can end an odyssey of medical tests and visits to multiple medical specialists. It also helps physicians better ...
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based in Durham, NORTH CAROLINA (USA)
Baebies was founded in 2014 by Richard West and Vamsee Pamula, following the successful development of digital microfluidics technology, the development and launch of multiple products, and the eventual sale of Advanced Liquid Logic to Illumina, ...
SEEKER - Newborn Screening Laboratory Solution
SEEKER is a newborn screening laboratory solution that performs multiple assays at the same time using just one punch from a newborn dried blood spot specimen. SEEKER is used to test thousands of babies each day around the world. ...
REQUEST QUOTEBaebies was founded in 2014 by Richard West and Vamsee Pamula, following the successful development of digital microfluidics technology, the development and launch of multiple products, and the eventual sale of Advanced Liquid Logic to Illumina, ...
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based in San Rafael, CALIFORNIA (USA)
Established in 1997, BioMarin is a world leader in developing and commercializing first- or best-in-class therapies for rare genetic diseases. We take pride in going where the science leads us, pioneering breakthrough treatments for debilitating and ...
Aldurazyme - Aldurazyme - Laronidase for MPS I
been shown to improve pulmonary function and walking capacity. ALDURAZYME has not been evaluated for effects on the central nervous system manifestations of the ...
REQUEST QUOTEEstablished in 1997, BioMarin is a world leader in developing and commercializing first- or best-in-class therapies for rare genetic diseases. We take pride in going where the science leads us, pioneering breakthrough treatments for debilitating and ...
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based in Budapest, HUNGARY
CycloLab is a worldwide recognized and all-around cyclodextrin research and development company, having the longest tradition and broadest circle of customers. Since 1972, a staff of internationally acknowledged experts have been working in the ...
CycloLab is a worldwide recognized and all-around cyclodextrin research and development company, having the longest tradition and broadest circle of customers. Since 1972, a staff of internationally acknowledged experts have been working in the ...
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