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galactosemia

Synonyms

Noun

1. inborn error of metabolism

Usage
Applied Therapeutics, Inc. SEC 10-K Report - TradingView
Why pharma is still far from a treatment for galactosemia - Clinical Trials Arena
Monosaccharides improve symptoms of an animal model for type III galactosemia, through the activation of the insulin pathway - ScienceDirect.com
FDA Issues CRL for Govorestat Application As Treatment for Classic Galactosemia - NeurologyLive
FDA Denies Approval of Govorestat for Classic Galactosemia - Endocrinology Advisor
Wait for galactosemia therapy goes on after FDA shuns Applied Therapeutics’ govorestat - Pharmaceutical Technology
Applied’s rare disease drug rejected by FDA - BioPharma Dive
Applied Therapeutics Receives Complete Response Letter from U.S. FDA Regarding New Drug Application for Govorestat for Classic Galactosemia - GlobeNewswire
Health and well‐being of maturing adults with classic galactosemia - Wiley Online Library
Applied Therapeutics Crashes After FDA Rejection of Rare Disease Drug - BioSpace
(PDF) Galactosemia: Towards Pharmacological Chaperones - ResearchGate
Contraindications to Breastfeeding - Centers for Disease Control and Prevention (.gov)
FDA Rejects Applied Therapeutics' Govorestat for Classic Galactosemia Treatment - Stock Titan
Sepsis caused by Phytobacter diazotrophicus complicated with galactosemia type 1 in China: a case report - BMC Infectious Diseases
US FDA declines to approve Applied Therapeutics' genetic disease drug - Reuters
Assessment of Long-Term Safety and Efficacy of Purple Sweet Potato Color (PSPC) and Myo- Inositol (MI) Treatment for Motor Related and Behavioral Phenotypes in a Mouse Model of Classic Galactosemia - Wiley Online Library
Applied Therapeutics Provides Regulatory Update on Govorestat for the Treatment of Classic Galactosemia - GlobeNewswire
FDA Action Alert: Applied Therapeutics Awaits Verdict on Rare Disease Therapy - BioSpace
Applied Therapeutics Reports Third Quarter 2024 Financial Results - GlobeNewswire
Biomarker discovery in galactosemia: Metabolomics with UPLC/HRMS in dried blood spots - Frontiers
Applied Therapeutics Drug Is Denied FDA Approval in Rare Inherited Metabolic Disease - MedCity News
Applied Therapeutics Stock Plummets After FDA Rejects Metabolic Disease Drug - Investopedia
APLT Stock Plunges 77% After FDA Issues CRL for Galactosemia Drug NDA - Yahoo Finance
(PDF) Acute and early developmental outcomes of children with Duarte galactosemia - ResearchGate
StockWatch: FDA Surprise Lifts Expectations for Applied’s Govorestat - Genetic Engineering and Biotechnology News
FDA declines approval for Applied Therapeutics' Galactosemia drug - Investing.com
Applied Therapeutics Provides Regulatory Update on Govorestat for the Treatment of Classic Galactosemia - Stock Titan
Applied Therapeutics' trial conduct scrutinized in FDA warning letter - Fierce Biotech
(PDF) Purpura fulminans in a newborn infant with galactosemia - ResearchGate
Applied Thera tanks, as FDA slaps CRL on govorestat NDA - The Pharma Letter
Complete Response Letter, Warning Letter and Shareholder Lawsuit Follow FDA Data Integrity Findings - JD Supra
Applied Therapeutics Stock Soars As FDA Scraps Panel Meeting For Its Inherited Metabolic Disorder Drug - Yahoo Finance
Applied Therapeutics: Strong Buy With AT-007 Nearing Approval For Galactosemia (APLT) - Seeking Alpha
(PDF) A Case of Classic Galactosemia Manifesting as Neonatal Early and Profound Indirect hyperbilirubinemia - ResearchGate
(PDF) The risk of classical galactosaemia in newborns with borderline galactose metabolites on newborn screening - ResearchGate
Why Is Applied Therapeutics Stock Trading Lower On Friday? - Applied Therapeutics (NASDAQ:APLT) - Benzinga
Galactosemia: Alterations in Sulfate Metabolism Secondary to Galactose-1-Phosphate Uridyltransferase Deficiency - Science | AAAS
Acute and early developmental outcomes of children with Duarte galactosemia - Fridovich‐Keil - 2022 - JIMD Reports - Wiley Online Library
Galactosemia Foundation Urges FDA to Incorporate Voice of the Patient in Review of Potential First Treatment for Children with Life-Altering Rare Disease - PR Newswire
(PDF) An Infant with Galactosemia: A Case Report - ResearchGate
Editorial: Inborn errors of carbohydrate metabolism - Frontiers
FDA extends review period for govorestat to treat classic galactosemia - contemporarypediatrics.com
(PDF) Assessment of galactose-1-phosphate uridyltransferase activity in cells and tissues - ResearchGate
New Resource for the Galactosemia Community Launches to Empower Families as They Navigate Lifelong Genetic Disease - Business Wire
Milk or no milk? Study fills long‑time knowledge gap on babies with genetic disorder - WSU Insider
Extreme neonatal hyperbilirubinemia, acute bilirubin encephalopathy, and kernicterus spectrum disorder in children with galactosemia - Nature
Low prevalence of classical galactosemia in Korean population - Nature
Figure 2. Interactions of arginine in the active site with enzyme and... - ResearchGate
The Leloir pathway of galactose metabolism. ADP, adenosine diphosphate;... - ResearchGate
Long-term speech and language developmental issues among children with Duarte galactosemia - Nature
Sanger sequencing confirmed that the patient was a compound... - ResearchGate
The Galactosemia Foundation and National Organization for Rare Disorders (NORD) Jointly Publish First "Voice of the Patient" Report to Help Inform the FDA and Researchers - PR Newswire
Biallelic GALM pathogenic variants cause a novel type of galactosemia - Nature
FDA Extends Review Period for Applied Therapeutics’ Govorestat by Three Months - BioSpace
High-Throughput Sequencing Reveals the Loss-of-Function Mutations in GALT Cause Recessive Classical Galactosemia - Frontiers
Multi-omics in classical galactosemia: Evidence for the involvement of multiple metabolic pathways - Wiley Online Library
Effects of temporary low-dose galactose supplements in children aged 5–12 y with classical galactosemia: a pilot study - Nature
Developmental Outcomes in Duarte Galactosemia | Pediatrics | American Academy of Pediatrics - AAP
Novel mRNA therapy restores GALT protein and enzyme activity in a zebrafish model of classic galactosemia - Wiley Online Library
World map of countries screening for galactosemia in neonates.⁹¹ - ResearchGate
Applied Therapeutics Seeks NDA for Rare Pediatric Disease Drug Despite Phase III Failure - BioSpace
Exploration of the Brain in Rest: Resting-State Functional MRI Abnormalities in Patients with Classic Galactosemia - Nature
Table 6 : Strategies for monitoring patients with classic galactosemia... - ResearchGate
Molecular characterization of novel and rare DNA variants in patients with galactosemia - Frontiers
Table 5 : Strategies for monitoring patients with classic galactosemia... - ResearchGate
Table 1 Clinical summary of patients with galactosemia and results of... - ResearchGate
Verbal Dyspraxia and Galactosemia - Nature
Galactokinase deficiency: lessons from the GalNet registry - Nature
Galactosemia…what is it? Family shares story of daughters rare disease - WLNS 6 News
Positive galactosemia newborn screening and following diagnostic... - ResearchGate
Fruit flies and milk sugar - Nature
Subfertility and growth restriction in a new galactose-1 phosphate uridylyltransferase (GALT) - deficient mouse model - Nature
Early and severe indirect hyperbilirubinemia as a manifestation of galactosemia - Nature
Fig. 1. Diagnostic Flowchart for Galactosemia Screening and Management.... - ResearchGate
Galactosemia Treatment Market Expected to Double by 2032, Projected Growth at 6.8% CAGR | Marketrese - PharmiWeb.com
Elevated Carbohydrate-Deficient Transferrin (CDT) and Its Normalization on Dietary Treatment as a Useful Biochemical Test for Hereditary Fructose Intolerance and Galactosemia | Pediatric Research - Nature
FDA decision delay for Applied Tx rare disease drug sends biotech's stock tumbling after hours - Fierce Biotech
Summary of reported galactose in various foods and ingredients. - ResearchGate
Applied Therapeutics Announces Partnership with Advanz - GlobeNewswire
FIGURE 1 (A) Number of male and female cases of galactosemia associated... - ResearchGate
Galactosemia: The good, the bad, and the unknown - Wiley Online Library
Fig. 1 The pedigree of the patient - ResearchGate
Applied Therapeutics to Present New Data on Prevention and Treatment of Galactosemia Complications at the Galactosemia Foundation Conference - Newswire :) Press Release Distribution
Table 2 Newborn screening testing strategies of respondent state labs - ResearchGate
Infant With Rare Disorder Saved After Lab-Tech Braves Boston Snow - ABC News
Mutations at the Galactose-1-P-Uridyltransferase Gene in Infants with a Positive Galactosemia Newborn Screening Test - Nature
Fertility preservation strategy in patients with galactosemia - ResearchGate
Galactose metabolism pathways; Leloir, oxidative and reductive galactose degradation pathways. - ResearchGate
AAFP Releases Position Paper on Breastfeeding - American Academy of Family Physicians | AAFP
US FDA extends review of Applied Therapeutics' genetic disease drug - Reuters
Congenital Cataracts - Consultant360
FDA extends review period for Applied Therapeutics’ govorestat NDA - Pharmaceutical Technology
Neonatal GALT gene replacement offers metabolic and phenotypic correction through early adulthood in a rat model of classic galactosemia - Wiley Online Library
Classical galactosaemia: novel insights in IgG N -glycosylation and N -glycan biosynthesis - Nature
Fig. 1. Frequencies of presenting symptoms and signs of studied cohort... - ResearchGate
A rare disease: Two local boys live with galactosemia - Manchester TImes
Towards individual prognostication in Classical Galactosemia AGEM - amsterdamumc.org
Bad to worse for Applied Therapeutics as FDA slaps partial clinical hold on rare drug - Fierce Biotech
Table 2 : Presentation data, GALT activity and mutation type of... - ResearchGate
Rare Disease Day 2024: Cystic Fibrosis to Galactosemia, know ways to manage rare diseases in newborns - India TV News
Foreign Language Equivalents
Foreign Language Equivalents of 'galactosemia'
Language Equivalent   Language Equivalent
French    Spanish
Misspellings - Typos
halactosemia,   galactoseia,   ga;actosemia,   ggalactosemia,   galctosemia,   galactosmia,   galacosemia,   gaalactosemia,   galactosemiia,   galactosemua,   falactosemia,   gaactosemia,   galactosemai,   galsctosemia,   glaactosemia,   galactpsemia,   glactosemia,   galactosenia,   galactosemmia,   galavtosemia,   galatosemia,   galactoesmia,   galactoosemia,   galactoseima,   galactosemis,   galactisemia,   galacyosemia,   galactose,ia,   galactossemia,   galacrosemia,   gakactosemia,   galactoemia,   aglactosemia,   gslactosemia,   galcatosemia,   galactosrmia,   galactoaemia,   galacttosemia,   galactosemi,   galactosmeia,   galactodemia,   galactsoemia,   galactoswmia,   galactosemoa,   galactosema,   galatcosemia,   galaactosemia,   gaalctosemia,   galaxtosemia,   gallactosemia,   galactoseemia,   galacctosemia,   alactosemia,   galactsemia,   galactosemiaa,   galacotsemia
Words Starting with Letter - 'A'
a a battery a billion
a bit a bit much a capella singing
a cappella singing a couple of a few
a fortiori a good deal a great deal
a horizon a hundred a hundred and one
a hundred thousand a hundred times a kempis
a la carte a la mode a level
a little a lot a million
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