A clinical study to evaluate the medicinal product in Patients with Propionic Acidemia
A Global, Phase 1/2, Open-label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia A Global, Phase 1/2, Open-label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, a ...
Propionic Acidemia- Propionic acidemia is an autosomal recessive disorder caused by loss-of-function mutations in PCCA or PCCB (and in rare instances, mutations in both PCCA and PCCB), resulting in the lossof enzymatic activity of the mitochondrial complex PCC MedDRA version: 21.0;Level: LLT;Classification code 10080615;Term: Propionic acidemia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] Propionic Acidemia- Propionic acidemia is an autosomal recessive disorder caused by loss-of-function ...
Product Name: mRNA-3927 INN or Proposed INN: N/A Other descriptive name: CX-016492 INN or Proposed INN: NA Other descriptive name: CX-017950 Product Name: mRNA-3927 INN or Proposed INN: N/A Other descriptive name: CX-016492 INN or Proposed INN: ...