Hasil Pencarian (2)
biotech | CAS: 1922968-73-7
Onasemnogene abeparvovec is an adeno-associated virus vector-based gene therapy that has been approved by the FDA in May 2019 for the treatment of infant patients (less than 2 years of age) with spinal muscular atrophy (SMA) and a specific mutation in the sur…
Kategori:
Adeno-associated Viral Vector TherapiesAmino Acids, Peptides, and ProteinsBiological Products
+7
Waktu ParuhThere is limited ph…
Vol. DistribusiThere is limited ph…
KlirensThere is limited ph…
Genetik
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small molecule | CAS: 1825352-65-5
Risdiplam is an orally bioavailable mRNA splicing modifier used for the treatment of spinal muscular atrophy (SMA).[L12615] It increases systemic SMN protein concentrations by improving the efficiency of _SMN2_ gene transcription. This mechanism of action is …
Kategori:
BCRP/ABCG2 SubstratesCytochrome P-450 CYP3A SubstratesCytochrome P-450 CYP3A4 Substrates
+11
Waktu ParuhThe terminal elimin…
Vol. DistribusiFollowing oral admi…
KlirensFor a 14.9kg patien…
Genetik
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