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Novel Adeno-associated Viral (AAV) Vectors To Treat Hereditary Methylmalonic Acidemia (MMA) Caused By Methylmalonyl-coA Mutase (MMUT) Deficiency Read more about Novel Adeno-associated Viral (AAV) Vectors To Treat Hereditary Methylmalonic Acidemia (MMA) Caused By Methylmalonyl-coA Mutase (MMUT) Deficiency US Application 63/080,337Filed on 2020-09-18
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DIAGNOSIS AND TREATMENT OF VEXAS, WITH MOSAIC MISSENSE MUTATIONS IN UBA1 Read more about DIAGNOSIS AND TREATMENT OF VEXAS, WITH MOSAIC MISSENSE MUTATIONS IN UBA1 US Application 63/059,107Filed on 2020-07-30
ABERRANT POST-TRANSLATIONAL MODIFICATIONS (PTMS) IN METHYL- AND PROPIONIC ACIDEMIA AND A MUTANT SIRTUIN (SIRT) TO METABOLIZE PTMS Read more about ABERRANT POST-TRANSLATIONAL MODIFICATIONS (PTMS) IN METHYL- AND PROPIONIC ACIDEMIA AND A MUTANT SIRTUIN (SIRT) TO METABOLIZE PTMS US Application 63/021,179Filed on 2020-05-07