Indication name: Methylmalonic Acidemia (MMA)
Methylmalonic Acidemia (MMA) – Market
outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020
To 2030 (Markets covered: US, Germany, France, Spain, United Kingdom, Italy,
Japan & China).
Methylmalonic acidemia/isolated
methylmalonic acidemia/methylmalonic aciduria/MMA refers to a group of
inherited conditions in which the body can’t breakdown certain parts of
proteins and fats. This leads to a build-up of toxic substances and bouts of
serious illness called decompensation events or metabolic crises. Isolated
methylmalonic acidemia is caused by changes in one of five genes: MMUT, MMAA,
MMAB, MMADHC, or MCEE. Methylmalonic acidemia with homocystinuria is caused by
mutations in the MMADHC, LMBRD1 and ABCD4 genes. Other forms of methylmalonic
acidemia are caused by changes in different genes.
Epidemiology- According to literature
review and Thelansis estimation, about 1/80,000-1/100,000 babies are born with
methylmalonic acidemia worldwide. In the United States, one study estimated
that about 1/90,000 babies are born with methylmalonic acidemia.
Competitive landscape of Methylmalonic
Acidemia (MMA) includes country specific approved as well as pipeline
therapies. Any asset/ product specific designation or review and Accelerated
Approval are being tracked and supplemented with analyst commentary.
KOLs insights of Methylmalonic Acidemia
(MMA) across 8 MM market from center of Excellence/ Public/ Private hospitals
participated in the study. Insights around current treatment landscape,
epidemiology, clinical characteristics, future treatment paradigm and Unmet
needs.
Methylmalonic Acidemia (MMA) Market
Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)
which Data Inputs with sourcing, Market Event and Product Event, Country
specific Forecast Model, Market uptake and patient share uptake, Attribute
Analysis, Analog Analysis, Disease burden and pricing scenario, Summary and
Insights.
S. No Companies Asset Status
1
Recordati Carglumic
acid Marketed in EU
2
ModernaTX, Inc. mRNA
3704 Phase I/II
3
HemoShear Therapeutics
HST-103 In pre-clinical
4
HemoShear Therapeutics
HST-104 In pre-clinical
5
HemoShear Therapeutics
methylmalonic acidemia In
pre-clinical
6
LogicBio LB-001 In pre-clinical
7 Selecta Biosciences SEL-302 In pre-clinical
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