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2. List of drugs granted breakthrough therapy designation

   en.wikipedia.org/wiki/List_of_drugs_granted...

   Treatment of adults with locally advanced, unresectable (stage III) non-small cell lung cancer (NSCLC) whose disease has not progressed during or following concurrent or sequential platinum-based chemoradiation therapy and whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test [2] Repotrectinib

3. Cipaglucosidase alfa - Wikipedia

   en.wikipedia.org/wiki/Cipaglucosidase_alfa

   Cipaglucosidase alfa, sold under the brand name Pombiliti, and used in combination with miglustat, is a medication used for the treatment of glycogen storage disease type II (Pompe disease). [4] [5] Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α-glucosidase ...

4. Avalglucosidase alfa - Wikipedia

   en.wikipedia.org/wiki/Avalglucosidase_alfa

   Avalglucosidase alfa is composed of the human GAA enzyme that is conjugated with a couple of bis-mannose-6-phosphate (bis-M6P) tetra-mannose glycans. [12] The bis-MGP of avalglucosidase alpha binds to the cation-independent mannose-6-phosphate receptor which is located on the skeletal muscles. [12]

5. Sanofi (SNY) Pompe Disease Drug Receives FDA's Approval - AOL

   www.aol.com/news/sanofi-sny-pompe-disease-drug...

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6. Non-small-cell lung cancer - Wikipedia

   en.wikipedia.org/wiki/Non-small-cell_lung_cancer

   The main treatment arms of phase 3 clinical trials providing immunotherapy in the first line for patients with non-small cell lung cancer. [59] Non-small cell lung cancer (NSCLC) cells expressing programmed death-ligand 1 (PD-L1) could interact with programmed death receptor 1 (PD-1) expressed on the surface of T cells, and result in decreased ...

7. Glycogen storage disease type II - Wikipedia

   en.wikipedia.org/wiki/Glycogen_storage_disease...

   Glycogen storage disease type II (GSD-II), also called Pompe disease, and formerly known as GSD-IIa or Limb–girdle muscular dystrophy 2V, is an autosomal recessive metabolic disorder [1] which damages muscle and nerve cells throughout the body.