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Qaagi - Book of Why

Causes

Effects

a factcausesPompe disease cases

by the lack of acid alpha - glucosidase activity ( GAA(passive) is caused byInfantile - onset Pompe disease

complete or partial deficiency of the(passive) is caused byHuman Pompe disease

the most significantinfluencingPompe Disease Drug

bear mutations homologous to thosecausehuman Pompe disease

indirect support for a model in whichcontributesto Pompe disease pathogenesis

with biologics program , ATB200 / AT2221 , which is making great steps in treating the genetic disorder(passive) is being ledPompe disease treatment

mutations in the gene encoding the enzyme alpha - glucosidase , or GAA , which results in a deficiency of GAA protein and leads to the accumulation of glycogen in an intracellular organelle known as the lysosome(passive) are caused byBoth forms of Pompe disease

the deficiency of the lysosomal enzyme acid(passive) is caused byPompe disease ( PD

a deficiency of lysosomal acid(passive) is caused byPompe disease ( PD

the deficiency of lysosomal acid(passive) is caused byPompe disease ... ( GAA

by cavitation(passive) caused bypump condition

a defective gene that results in a deficiency of an enzyme , acid alpha - glucosidase ( GAA(passive) is caused byPompe disease

mutations in the gene encoding the lysosomal enzyme alpha - glucosidase , or GAA , which results in a deficiency of GAA protein and leads to the accumulation of glycogen(passive) is caused byPompe disease

mutations in the gene encoding the lysosomal enzyme alpha - glucosidase , or GAA , which results in a deficiency of GAA protein and leads to the intracellular accumulation of glycogen(passive) is caused byPompe disease

mutations in the gene encoding the lysosomal enzyme alpha - glucosidase , or GAA , which results in a deficiency of GAA protein and leads to an intracellular accumulation of glycogen(passive) is caused byPompe disease

a defective gene that results in a deficiency of an enzyme , acid alpha - glucosidase(passive) is caused byPompe disease

a mutation in the GAA gene that leads to a deficiency in an enzyme called acid alpha - glucosidase(passive) is caused byPompe disease

glycogen accumulation as a result of deficiency of the lysosomal enzyme acid alpha glucosidase ( GAA(passive) is caused byPompe disease

a deficiency in the enzyme acid alpha - glucosidase ( GAA(passive) is caused byPompe disease

mutations in acid alpha glucosidase ( GAA ) that causes accumulation of(passive) is caused byPompe disease

a defective gene that results in a deficiency of an enzyme(passive) is caused byPompe disease

by a defective gene that results in a deficiency of an enzyme(passive) is caused byPompe disease

the absence or marked deficiency of the lysosomal enzyme acid alpha - glucosidase ( GAA(passive) is caused byPompe disease

pathogenic variations in the acid alpha - glucosidase ( GAA ) gene(passive) is caused byPompe disease

a deficiency of the lysosomal acid alpha glucosidase ( GAA(passive) is caused byPompe disease

A deficiency of the enzyme acid alpha - glucosidaseleadsto Pompe disease

mutations in the acid alpha - glucosidase ( GAA ) gene that leads to either the complete absence or a less effective version of the GAA enzyme(passive) is caused byPompe disease

a deficiency of the enzyme alpha - glucosidase ( GAA ) , an enzyme that at normal levels will breakdown glycogen in the body(passive) is caused byPompe disease

by the deficiency of acid alpha - glucosidase ( GAA(passive) caused byPompe disease

mutations of the GAA gene(passive) is caused byPompe disease

the deficiency of acid - alpha - glucosidase ( GAA ) in muscle(passive) is caused byPompe disease

mutations in the GAA gene , which contains the information necessary to make the enzyme acid alpha - glucosidase(passive) is caused byPompe disease

a deficiency of the enzyme alpha - glucosidase ( GAA ) , an enzyme that at normal levels will break down glycogen in the body(passive) is caused byPompe disease

an absence or deficiency of the lysosomal enzyme acid(passive) is caused byPompe disease

a deficiency of the lysosomal enzyme , GAA(passive) is caused byPompe disease

A mutation in the GAA gene causes glycogen breakdownleadingto Pompe disease

a deficiency of the lysosomal acid alpha glucosidase ( GAA ) , responsible for breaking down glycogen(passive) is caused byPompe disease

absence of the lysosomal enzyme acid alpha - glucosidase(passive) is caused byPompe disease

mutation of the GAA gene(passive) is caused byPompe disease

muscle weakness and trouble breathingcausesmuscle weakness and trouble breathing

in deathcan resultin death

to cardiac and/or respiratory failureleadsto cardiac and/or respiratory failure

protein losscan causeprotein loss

usuallyresultsusually

Renal hematuria(passive) is caused byRenal hematuria

by an enzyme deficiency that leads to accumulation of glycogen in cellscausedby an enzyme deficiency that leads to accumulation of glycogen in cells

progressive , often life - threatening musculoskeletal , respiratory , and cardiac symptomscausesprogressive , often life - threatening musculoskeletal , respiratory , and cardiac symptoms

progressive , debilitating , and often life - threatening musculoskeletal , respiratory , and cardiac symptomscausesprogressive , debilitating , and often life - threatening musculoskeletal , respiratory , and cardiac symptoms

muscle weakness even in early diagnosed patientscausesmuscle weakness even in early diagnosed patients

severe disabilitycausessevere disability

to deathleadsto death

lysosomal glycogen accumulation in skeletal muscle and heartcauseslysosomal glycogen accumulation in skeletal muscle and heart

oftenresultsoften

to heart failurecan leadto heart failure

progressive muscle weakness and a great deal of paincausesprogressive muscle weakness and a great deal of pain

muscle weakness and trouble breathingcausesmuscle weakness and trouble breathing

itchingcan causeitching

the accumulation of glycogen in muscle tissuecausesthe accumulation of glycogen in muscle tissue

muscle weakness , impaired gait , and muscle painmainly causesmuscle weakness , impaired gait , and muscle pain

from a deficiency or absence of the lysosomal enzyme acid alpha glucosidase ( GAAresultsfrom a deficiency or absence of the lysosomal enzyme acid alpha glucosidase ( GAA

muscle wasting in my arms and legshas causedmuscle wasting in my arms and legs

from a genetic mutation in the GAA generesultsfrom a genetic mutation in the GAA gene

the diaphragmatic weakening(passive) caused bythe diaphragmatic weakening

the accumulation of glycogen , used to store energy , in muscle tissuecausesthe accumulation of glycogen , used to store energy , in muscle tissue

in human cellscausedin human cells

alsocan ... resultalso

from mutations in the gene encoding the glycogen - degrading lysosomal enzyme acid alpharesultsfrom mutations in the gene encoding the glycogen - degrading lysosomal enzyme acid alpha

What Is Used In Male Enhancement PillscausingWhat Is Used In Male Enhancement Pills

in pressurized sacrificed perfusionresultingin pressurized sacrificed perfusion

in a Golgi - based glycosylation deficit in human induced pluripotent stem cell - derived cardiomyocytesresultsin a Golgi - based glycosylation deficit in human induced pluripotent stem cell - derived cardiomyocytes

a record global category womenseta record global category women

damagecauseddamage

for neonates and infants with a variety of pathologiesdesignedfor neonates and infants with a variety of pathologies

to run over a broad range of performance and deliver outstanding suction liftdesignedto run over a broad range of performance and deliver outstanding suction lift

to operate over a broad range of performance while delivering outstanding suction liftdesignedto operate over a broad range of performance while delivering outstanding suction lift

The easy - to - use , one - touch(passive) is designedThe easy - to - use , one - touch

for use on diesel drive pumpsdesigned specificallyfor use on diesel drive pumps

for continuous pumping of highly abrasive and corrosive slurriesdesignedfor continuous pumping of highly abrasive and corrosive slurries

FreshVac Technology - The easy - to - use , one - touch(passive) is designedFreshVac Technology - The easy - to - use , one - touch

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