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  1. 1 giorno fa · A multidisciplinary research team has developed a discovery platform to probe the function of genes involved in metabolism — the sum of all life-sustaining chemical reactions. The investigators used the new platform, called GeneMAP (Gene-Metabolite Association Prediction), to identify a gene necessary for mitochondrial choline transport.

  2. 5 ore fa · Join us on WhatsApp. A miracle new treatment approved yesterday is set to change the life of Butterfly girl Isla Grist who suffers in agony every day. Scotland legend Graeme Souness' wee pal Isla ...

  3. 1 giorno fa · To discover functions for "orphan" transporters and enzymes — proteins with unknown substrates — the researchers developed the GeneMAP discovery platform. "What’s exciting about this study is its interdisciplinarity — the combination of genomics and metabolism to identify a long-sought mitochondrial choline transporter," Gamazon said.

  4. 2 ore fa · -- Neurotech International filed for orphan drug designation for neurological drug NTI164 with the US Food and Drug Administration. NTI164 aims to treat children and adults diagnosed with pediatric...

  5. 5 ore fa · The Orphan Drug Act defines a rare disease as a disease or condition that affects less than 200,000 people in the United States. PANDAS/PANS is a rare neurological disorder predominately in ...

  6. 5 ore fa · The average price for an orphan drug is US$32,000 per year per patient and government approvals to market are fast tracked. Today’s announcement is further evidence that NTI is delivering, and aligns well with one of the key reasons we Invested in NTI. NTI will need to move quickly to establish its presence in the market.

  7. 1 giorno fa · SELLAS Life Sciences Group, Inc. gab bekannt, dass die Europäische Kommission auf der Grundlage einer positiven Stellungnahme der Europäischen Arzneimittelagentur (EMA) SLS009, einem neuartigen und hochselektiven CDK9-Inhibitor, den Orphan Drug Designation (ODD) für die Behandlung von akuter myeloischer Leukämie (AML) erteilt hat.

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