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mucopolysaccharidosis

Synonyms

Noun

1. genetic disease

2. genetic disorder, genetic abnormality, genetic defect, congenital disease, inherited disease, inherited disorder, hereditary disease, hereditary condition

Usage
Our Origin Story - BioMarin Pharmaceutical
TFEB, FOXO3 and TLR4 in resveratrol-induced autophagy in a mucopolysaccharidosis IIIB mouse model - Nature
Case Report: Compound heterozygous mutations in the IDUA gene causing mucopolysaccharidosis type I with uterine developmental abnormality - Frontiers
FDA Review Extension for Tividenofusp Alfa in MPS II (Hunter Syndrome): Regulatory and Clinical Implications - Vera Health
FDA issues CRL for RGX-121 gene therapy in mucopolysaccharidosis type II - Contemporary Pediatrics
Immusoft Receives FDA Rare Pediatric Disease Designation for ISP-002 to treat Mucopolysaccharidosis Type II - PR Newswire
A Rare Case of Mucopolysaccharidosis Presenting With Dysostosis Multiplex and Preserved Intelligence in a Seven-Year-Old Girl From Northeast India - Cureus
FDA Places Clinical Hold on Gene Therapies for MPS I and MPS II - MPR - Medical Professionals Reference
Cell Therapy Weekly: First IND Cleared for AAV-based Cancer Therapy - RegMedNet
Brain-penetrant enzyme replacement therapy improves biomarkers in mucopolysaccharidosis type II - 2 Minute Medicine
Experimental Drug Shows Promise for Rare Genetic Disorder - Northwestern University
Hunter Syndrome Therapy That Crosses Blood-Brain Barrier Shows Promise - Neurology Today
JCR Pharmaceuticals Presents Long-Term Clinical Data on Pabinafusp Alfa for the Treatment of Mucopolysaccharidosis Type II (MPS II) at ICIEM 2025 - Business Wire
Ultragenyx’s gene therapy shows sustained improvements in MPS IIIA - Clinical Trials Arena
Renowned UNC Pediatric Biochemical Geneticist Leads First Study on a Novel Treatment for Hunter Syndrome - UNC Health
Turning Point for Hunter Syndrome? - MedPage Today
Comparison of machine learning models for mucopolysaccharidosis early diagnosis using UAE medical records - Nature
JCR Pharmaceuticals’ Research Presentations at the 18th International Symposium on MPS and Related Lysosomal Diseases 2026 Showcase Data from Its Investigational Treatments for Lysosomal Storage Disorders - Business Wire
Ultrasound-guided one-point puncture lumbosacral plexus block combined with laryngeal mask airway general anesthesia for thigh amputation in a patient with mucopolysaccharidosis: a case report - Frontiers
Efficacy and safety of a biosimilar laronidase versus the reference laronidase in patients with mucopolysaccharidosis type I - Nature
Denali’s Hunter Syndrome Candidate in the Spotlight After REGENXBIO Rejection - BioSpace
JCR Pharmaceuticals to Present at the 18th International Symposium on MPS and Related Lysosomal Diseases 2026 - Business Wire
JCR Pharmaceuticals’ Research Presentations at WORLDSymposium™ 2026 Showcase Data from Its Investigational Treatments for Lysosomal Storage Disorders - Business Wire
FDA Bumps Back PDUFA for REGENXBIO's MPS II Gene Therapy RGX-121 - CGTLive®
Child Neurology: Mucopolysaccharidosis IIID - Neurology® Journals
FDA Fast Track Designation Granted to ISP-001: Engineered B Cell Therapy for MPS I Shows Early Safety and Functional Gains - Vera Health
Immusoft Secures FDA Orphan Drug Designation for ISP-002 in MPS II, a Progressive Lysosomal Storage Disease - PR Newswire
Mucopolysaccharidosis type II zebrafish model exhibits early impaired proteasomal-mediated degradation of the axon guidance receptor Dcc - Nature
Patients Treated With REGENXBIO's MPS II Gene Therapy RGX-121 Sustain 82% Median Reduction in CSF HS D2S6 Levels Through 1 Year - CGTLive®
An immune deficient mouse model for mucopolysaccharidosis IIIA (Sanfilippo syndrome) - Nature
Disease pathology signatures in a mouse model of Mucopolysaccharidosis type IIIB - Nature
JCR Pharmaceuticals to Present at the 22nd Annual WORLDSymposium™ 2026 - Business Wire
Physical therapy eases pain, improves life for Sanfilippo kids: Study - Sanfilippo Syndrome News
Mid-Michigan Matters: ‘Childhood Alzheimer’s’ fundraising for a treatment - WILX
Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy - Nature
Long term follow-up after haematopoietic stem cell transplantation for mucopolysaccharidosis type I-H: a retrospective study of 51 patients | Bone Marrow Transplantation - Nature
Hurler Syndrome (Mucopolysaccharidosis Type 1): A Case Report - Cureus
Evidence of epigenetic landscape shifts in mucopolysaccharidosis IIIB and IVA - Nature
Recent trends in mucopolysaccharidosis research - Nature
A novel mucopolysaccharidosis type II mouse model with an iduronate-2-sulfatase-P88L mutation - Nature
Mature neurons from iPSCs unveil neurodegeneration-related pathways in mucopolysaccharidosis type II: GSK-3β inhibition for therapeutic potential - Nature
Lysosomal and network alterations in human mucopolysaccharidosis type VII iPSC-derived neurons - Nature
FDA Halts Two Gene Therapy Trials After Child Develops Brain Tumor - MedPage Today
In a Major Breakthrough for Cell and Gene Therapy, Immusoft Has Re-dosed a Patient with ISP-001 in Its Lead Program for MPS I - PR Newswire
Natural history of cardiac findings in mucopolysaccharidosis type I: report from an international registry - Cambridge University Press & Assessment
Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I - Nature
FDA extends review of Regenxbio's MPS II gene therapy to assess longer-term data - FirstWord Pharma
Late‐onset mucopolysaccharidosis type IIIA mimicking Usher syndrome - Wiley Online Library
FDA Approves Tividenofusp alfa-eknm, Targeting Neurologic Manifestations of Hunter Syndrome - Pharmacy Times – Pharmacy Practice News and Expert Insights
FDA rejects Regenxbio's Hunter syndrome gene therapy - FirstWord Pharma
Treatment of mucopolysaccharidosis type II (Hunter syndrome): results from a systematic evidence review - Nature
This rare disease destroys a child's ability to walk and speak. Could a new drug help? - NBC News
Mucopolysaccharidosis 6 and gene therapy: present and future - Ricerca Malattie Genetiche Rare
Oral trehalose improves histological and behavior symptoms of mucopolysaccharidosis type II in iduronate 2-sulfatase deficient mice - Nature
Structural and mechanistic insight into the basis of mucopolysaccharidosis IIIB - PNAS
Ultragenyx Puts BLA for MPS IIIA Gene Therapy UX111 in Front of FDA Again - CGTLive®
N-glycan-modified α-L-iduronidase produced by transgenic silkworms ameliorates clinical signs in a Japanese macaque with mucopolysaccharidosis I | Communications Medicine - Nature
Atypical Neuroimaging Findings in a Patient With Mucopolysaccharidosis Type VII (Sly Syndrome) - Cureus
Association between mucopolysaccharidosis Type VII and hydrops fetalis - Wiley & Sons
Modified umbilical cord-blood transplantation for pediatric patients with mucopolysaccharidosis - Nature
FDA freezes two Regenxbio gene therapy programmes after safety signal - FirstWord Pharma
FDA details rationale for rejecting rare disease gene therapy from Regenxbio - Fierce Biotech
Airway distortion in mucopolysaccharidosis - Springer Nature Link
Teaching NeuroImages: Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome) in a previously undiagnosed infant - Neurology® Journals
Mucopolysaccharidosis (MPS): Review of the literature and case series of five pediatric dental patients - Wiley Online Library
Shutdown of ER-associated degradation pathway rescues functions of mutant iduronate 2-sulfatase linked to mucopolysaccharidosis type II - Nature
Denali Therapeutics Announces FDA Review Extension of BLA - GlobeNewswire
Mucopolysaccharidosis Type I: The Importance of Early Diagnosis for Adequate Treatment - Cureus
Liver-Directed Adeno-Associated Virus–Mediated Gene Therapy for Mucopolysaccharidosis Type VI - NEJM Evidence
FDA rejects Regenxbio’s rare-disease gene therapy - STAT
Single-cell RNA sequencing reveals important role of monocytes and macrophages during mucopolysaccharidosis treatment - Nature
FDA delays decision on Denali's Hunter syndrome therapy by three months - FirstWord Pharma
Teen’s acute psychotic disorder leads to Sanfilippo A diagnosis –... - Sanfilippo Syndrome News
Normalization of glycosaminoglycan-derived disaccharides detected by tandem mass spectrometry assay for the diagnosis of mucopolysaccharidosis - Nature
Mucopolysaccharidosis Type IIIA Presenting as Hypertrophic Cardiomyopathy | Circulation: Heart Failure - American Heart Association Journals
Arterial pathology in canine mucopolysaccharidosis-I and response to therapy - Nature
Alpha-L-iduronidase deficiency: A novel mutation resulting in severe early presentation of Mucopolysaccharidosis type I and literature review of the molecular basis - Wiley Online Library
Visual impairment in mucopolysaccharidosis VI - Magalhães - 2023 - JIMD Reports - Wiley Online Library
Relationship Between Attention and Corpus Callosum Volumes in Mucopolysaccharidosis Type I - Neurology Advisor
A case report of a patient with mucopolysaccharidosis type II - Elsevier
Corneal Clouding Stays Stable in Most Undergoing Mucopolysaccharidosis Treatment - Ophthalmology Advisor
Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII mice - PNAS
Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA - Wiley Online Library
Study Gives Parents' Views of Caring for Kids with Mucopolysaccharidosis - Sanfilippo Syndrome News
A 1-year and 4-month-old child with mucopolysaccharidoses type II: A clinical case report from Ethiopia - Wiley Online Library
Audiometric evaluation in individuals with mucopolysaccharidosis - Elsevier
Chronic subdural hematoma associated with mucopolysaccharidosis type III B (Sanfilippo’s syndrome B) - Springer Nature Link
Denali: Maintaining Strong Buy Based On DNL126 Expansion As MPS IIIA Treatment (DNLI) - Seeking Alpha
Structural study on mutant α-l-iduronidases: insight into mucopolysaccharidosis type I | Journal of Human Genetics - Nature
Efficient CRISPR/Cas9 nickase-mediated genome editing in an in vitro model of mucopolysaccharidosis IVA - Nature
Neuroimaging Findings in Patients with Mucopolysaccharidosis: What You Really Need to Know | RadioGraphics - RSNA Journals
Phenotypic expansion of ARSK -related mucopolysaccharidosis - Wiley Online Library
Anakinra in Sanfilippo syndrome: a phase 1/2 trial - Nature
The Newborn Screening Mucopolysaccharidosis Type I (MPS I) Pilot Program - RTI International
JCR Pharmaceuticals Initiates First Dosing in Phase I Clinical Trial of JR-441 for Mucopolysaccharidosis Type IIIA - BioSpace
GC Biopharma files for Korea approval of ICV Hunter syndrome therapy - koreabiomed.com
Mutational and structural analysis of Japanese patients with mucopolysaccharidosis type II - Nature
University of Manchester Launches MPS II Gene Therapy Trial, Picking Up Where AVROBIO Left Off - CGTLive®
A Novel Mutation in the NAGLU (N-Acetyl-Alpha-Glucosaminidase) Gene Associated With Mucopolysaccharidosis Type III-B in a Saudi Girl - Cureus
Hunter Syndrome (Mucopolysaccharidosis II): Diagnosis, Genetic Testing, Treatment, and Referral - Consultant360
Foreign Language Equivalents
Foreign Language Equivalents of 'mucopolysaccharidosis'
Language Equivalent   Language Equivalent
French    Spanish
Misspellings - Typos
mucopolysaccharifosis,   ,ucopolysaccharidosis,   mucopolysacchardiosis,   mucoolysaccharidosis,   mucpoolysaccharidosis,   muvopolysaccharidosis,   mucopolysaccharidsis,   mucppolysaccharidosis,   muxopolysaccharidosis,   mucopolysaccharidoais,   micopolysaccharidosis,   mycopolysaccharidosis,   muocpolysaccharidosis,   mucopolysacchaidosis,   mucopolysaccharidosus,   mucopolsaccharidosis,   mucopplysaccharidosis,   mucooolysaccharidosis,   mucoplysaccharidosis,   mucopolysacchharidosis,   mucopolysaccharidsois,   mucopolysccharidosis,   nucopolysaccharidosis,   mucopolysaccharidodis,   mmucopolysaccharidosis,   mucopolysaccharridosis,   mucopolysacchariidosis,   mucopolysacchaaridosis,   mucopolydaccharidosis,   mucopokysaccharidosis,   mucopollysaccharidosis,   muco[olysaccharidosis,   mucopolysacchsridosis,   mucopolysacharidosis,   mucopolysaccharodosis,   mucopolysaccharidosid,   mucopolysacchairdosis,   mucopoysaccharidosis,   mucopoolysaccharidosis,   mucopolysaxcharidosis,   mucopolysaccharidoss,   mucopoltsaccharidosis,   mucopolysachcaridosis,   muucopolysaccharidosis,   mucopolysaccaridosis,   mucopolysaccharidosiss,   mucopolysaccharidisis,   mucopolysacchraidosis,   mucopolysaccharidosos,   mucopolysacchariosis,   mucopolysacvharidosis,   mucopolysaaccharidosis,   mucopolysacchaeidosis,   mucopolysacxharidosis,   mucopolyssccharidosis,   muccopolysaccharidosis,   mucopolyssaccharidosis,   umcopolysaccharidosis,   mucopolysacchariodsis,   mucipolysaccharidosis,   mucopolysaccharidossi,   mucopolyaccharidosis,   ucopolysaccharidosis,   mucooplysaccharidosis,   mucopolysacccharidosis,   mucopolysaccharidosia,   mucopolysacchardosis,   mucopolyaaccharidosis,   mucopolysacccharidosis,   mucopolyscacharidosis,   mucopolysacchariddosis,   mucopoylsaccharidosis,   mucopolysaccharidoosis,   mucopolysaccharidosiis,   mucopolysaccharisosis,   mucopilysaccharidosis,   mucopolyasccharidosis,   muopolysaccharidosis,   mucopolusaccharidosis,   mucopolysaccharidpsis,   mucoopolysaccharidosis,   mucopolysaccgaridosis,   mucopolysaccahridosis,   mcuopolysaccharidosis,   mucopolysacchatidosis,   mucopolsyaccharidosis,   mucopolysaccharudosis,   mucopolysaccharidoiss,   mucoploysaccharidosis,   mucpolysaccharidosis,   mucopolysacchridosis,   mucopolysacharidosis,   mucopolysaccharidois,   mucopolysavcharidosis,   mucoppolysaccharidosis,   mcopolysaccharidosis,   mucopolysaccharidosi,   mucopo;ysaccharidosis,   mucopolysaccharidossis,   mucopolyysaccharidosis,   mucopolysaccjaridosis
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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