Sirio (carbidopa/melevodopa) / Chiesi, Ligand - LARVOL DELTA

Online Platform for SAMD9 and SAMD9L Variant Annotation and Phenotype Correlation (ASH 2022) - "Moreover, 60% (24/41) of SAMD9Lmut cases with SGR (UPD7q and somatic SAMD9/9Lmut) were associated with germline mutations affecting only 4 aa residues (H880, R986, R1281, and V1512)...Our study (www.stjude.org/samd9) represents the first effort to develop a genotype-phenotype database of SAMD9/9L syndromes which will allow investigators to deposit variants and compare them with published and unpublished data. In newly discovered predisposition syndromes, databases like the one presented here, will be instrumental in gathering crucial clinical data for variant interpretation, identify new phenotypic and prognostic correlates, and improving patients' clinical care."

Aplastic Anemia • Ataxia • Gastrointestinal Disorder • Glomerulonephritis • Immunology • Movement Disorders • Myelodysplastic Syndrome • Nephrology • Renal Disease

L-Dopa Pharmacokinetic Profile with Effervescent Melevodopa/Carbidopa versus Standard-Release Levodopa/Carbidopa Tablets in Parkinson's Disease: A Randomised Study. (PubMed) - "V1512 provides a more reliable L-dopa pharmacokinetic profile versus standard-release L-dopa/carbidopa, with less drug accumulation and less variability. This trial is registered with ClinicalTrials.gov NCT00491998."

Journal • Biosimilar • Parkinson's Disease

A systematic review on the clinical experience with melevodopa/carbidopa fixed combination in patients with Parkinson disease. (PubMed, Minerva Med) - "Data from literature have shown that melevodopa/carbidopa in a highly soluble formulation as effervescent oral tablets is effective in improving control of motor complications in PD patients compared to conventional levodopa. Further research is needed to confirm this evidence in large controlled trials and to explore if melevodopa/carbidopa may have a potential role in the earlier phases of the disease or in special PD populations."

Clinical • Journal • Review