Press Release: ASCO Names Advance of the Year: Progress in Treating Rare Cancers

Progress Report Debuts ASCO’s Research Priorities for the Cancer Community, Emphasizes Impact of Federal Funding FOR IMMEDIATE RELEASE January 31, 2019 Ashley Yum 571-483-1376 [email protected]   ALEXANDRIA, Va. — Over the…

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Results from Phase II/III Trial Bring Hope for FDA Approval for New Niemann-Pick Disease Type C Treatment

Niemann-Pick Disease is a genetic lysosomal storage disease. Lipids accumulate in the lysosomes of cells, causing debilitating symptoms. There are three forms of the disease- A, B, and C. Each…

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Yeast May be the Key to Uncovering Treatments for Homocystinuria and Other Metabolic Conditions

There is a great unmet medical need for patients living with metabolic disorders. There are few approved treatments for these conditions and many are fatal. These diseases include phenylketonuria, homocystinuria, tyrosinemia, and maple syrup…

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