Moderna previously received Fast Track designation for its methylmalonic acidemia program, which is now recruiting patients for a Phase 1/2 clinical study.
We are committed to building a broad pipeline of product candidates to capture the full value of GeneRide, beginning with our lead product candidate, LB-001 for the treatment of methylmalonic acidemia (MMA), a rare and life-threatening pediatric disease.
(6) The conference established rules for interpreting fetal heart rate (FHR) tracings from EFM: Category I (normal), Category II (indeterminate) which was not predictive of metabolic acidosis, and Category III (abnormal) which was associated with abnormal fetal acid-base status and the potential for metabolic acidemia. The group consensus was that EFM was a test of the current acid-base status of the fetus and was not predictive of adverse birth outcomes such as cerebral palsy.
These include methylmalonic academia or MMA (mRNA-3704 with an open IND for a Phase 1/2 study); Propionic Acidemia or PA (mRNA-3927 in IND-enabling GLP toxicology studies); and Phenylketonuria or PKU (mRNA-3283 in IND-enabling GLP toxicology studies).
The disorders of intracellular cobalamin metabolism that are inherited in an autosomal recessive manner result from the deficient synthesis of AdoCbl and MeCbl derived from Vitamin B12; the types of disorders includes: cblA, cblB, cblC, cblD, etc., depending on the pathogenic genes.[1],[2] The cblD disease (MIM# 277410) caused by mutations in the MMADHC gene contains three subtypes: cblD-isolated methylmalonic acidemia (MMA), cblD-isolated homocystinuria (HC) and cblD-MMA/HC (combined MMA and HC).[1] We herein reported the Chinese patient with cblD disease attributable to a novel MMADHC mutation related to translation reinitiation.
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