The prevalence of MMA is estimated to be 1 in 50,000 to 100,000 in North America and at 1/26,000 in China. The pooled point estimates of the detection rates were 0.79, 1.12, 1.22, and 6.04 per 100,000 newborns in Asia-Pacific, Europe, North America, and MENA, respectively. In Europe, the highest estimated cases are being reported in Italy with 4.40/100,000 newborns cases in comparison to other EU5 countries
Comprehensive insight on patient segmentation based on forms (isolated MMA and combined MMA), Mutations (MUT, MMAA, MMAB, MMADHC, and MCEE ), severity level (Mild and Severe) has been provided into the epidemiology (Incidence & Prevalence) section of the Methylmalonic academia and its treatment in the 8 MM countries, covering the United States, EU5 (Germany, Spain, France, Italy, UK), Japan, and China
Moderna Therapeutics is leading the competitive pipeline for methylmalonic academia with a Phase II drug candidate (mRNA-3704). Apart from Moderna’s therapeutic candidate, there is not much activity in the clinical stages for this indication. However, there are several players like HemoShear Therapeutics, LogicBio Therapeutics, and Selecta Biosciences who are active in the pre-clinical space with their products. The innovative mechanisms being followed by pipeline therapies include gene expression stimulation, mTOR kinase inhibition; cyclin inhibition; protein kinase inhibition, and T cell inhibition
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