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Lysosomal Storage Disorders Suppliers & Manufacturers
8 companies found
based inBethesda, 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 ...
Protein misfolding is the root of many diseases, including lysosomal storage disorders and neurodegenerative ...
based inMonmouth Junction, NEW JERSEY (USA)
MedChemExpress (MCE) offers a wide range of high-quality research chemicals and biochemicals (novel life-science reagents, reference compounds and natural compounds) for scientific use. We have professionally experienced and friendly staff to meet ...
based inRostock, 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 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 ...
based inBudapest, 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 ...
based inDurham, 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 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. ...
based inSan 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 (laronidase) is indicated for patients with Hurler and Hurler-Scheie forms of mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms. The risks and benefits of treating mildly affected ...
based inNaarden, NETHERLANDS
Azafaros has leveraged on decades of discovery by professors Hans Aerts, Hermen Overkleeft, and Stan van Boeckel at Leiden University and the Academic Medical Center in the Netherlands. Expanding on their research and led by highly experienced ...
Azafaros B.V. is pioneering the development of innovative oral small molecule compounds addressing the pathological effects of metabolite accumulation in rare metabolic disorders. Their lead compound, nizubaglustat, leverages unique ...
based inOxford, 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 ...
Niemann-Pick C disease (NPC) is a lysosomal storage disorder which affects the brain and multiple other ...