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Rare Disease Day 2017 With A Commitment To Provide New Insights Into Rare Diseases Services In Netherlands
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Manufactured by:MorphoSys AG based inPlanegg, GERMANY
Expanded Access to our Investigational Medicinal Products Policy. At MorphoSys, our mission is to make innovative biopharmaceuticals to improve the lives of patients suffering from serious diseases. Before these and all other investigational medicinal products can be made broadly available, the U.S. Food and Drug Administration (FDA) and other health authorities around the world require that ...
by:Azafaros based inNaarden, NETHERLANDS
Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized by lysosomal dysfunction, most of which are inherited as autosomal recessive traits. These disorders are individually rare but collectively affect 1 in 5,000 live births. They typically present in infancy and childhood, although adult-onset forms also occur. Most LSDs have a progressive neurodegenerative ...
by:Azafaros based inNaarden, NETHERLANDS
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 dual-mode action, making it a promising candidate for modifying diseases irrespective of patient genotypes. Notably effective against conditions such as GM1 and GM2 ...