318 results on '"mucopolysaccharidoses"'
Search Results
2. Patent Issued for Methods to treat mucopolysaccharidosis type II or deficiency in iduronate-2-sulfatase using a recombinant adeno-associated virus (AAV) vector encoding iduronate-2-sulfatase (USPTO 12121567).
3. New Gene Therapy Research Has Been Reported by a Researcher at Department of Otolaryngology (Otorhinolaryngological Problems in Mucopolysaccharidoses: A Review of Common Symptoms in a Rare Disease).
4. Patent Application Titled "Targeted Delivery Of Therapeutic Enzymes" Published Online (USPTO 20240350653).
5. New Findings from Ann and Robert H. Lurie Children's Hospital of Chicago Describe Advances in Mucopolysaccharidoses (Newborn Screening for Mucopolysaccharidosis Type Ii: Lessons Learned).
6. Study Findings on Mucopolysaccharidosis II Discussed by a Researcher at Takeda Development Center Americas Inc. (A Systematic Literature Review on the Global Status of Newborn Screening for Mucopolysaccharidosis II).
7. Data on Mucopolysaccharidoses Reported by Michael Hocquemiller and Colleagues (Tandem mass spectrometry-based assay for heparan-N-sulphatase in paediatric CSF: A potential pharmacodynamic biomarker for mucopolysaccharidosis type IIIA therapy).
8. Department of Medicine Researchers Publish Findings in Mucopolysaccharidoses [Intracranial tumor in a patient with mucopolysaccharidosis type 1 (Scheie syndrome): An extremely rare combination].
9. Studies Conducted at MacKay Memorial Hospital on Mucopolysaccharidoses Recently Published (Advancements in Otorhinolaryngological Management of Mucopolysaccharidosis: A Comprehensive Review).
10. Findings from University of North Carolina Chapel Hill Reveals New Findings on Mucopolysaccharidosis II (Evaluation of Early Treatment With Intravenous Idursulfase and Intrathecal Idursulfase-it On Cognitive Function In Siblings With...).
11. New Mucopolysaccharidoses Study Findings Have Been Reported by Researchers at Shanghai Health Development Research Center (Health service utilization, economic burden and quality of life of patients with mucopolysaccharidosis in China).
12. Nebulized and intravenous enzyme replacement therapy in mice with mucopolysaccharidosis type II.
13. Findings from University of Gdansk Advance Knowledge in Mucopolysaccharidoses (Mucopolysaccharidosis-Plus Syndrome: Is This a Type of Mucopolysaccharidosis or a Separate Kind of Metabolic Disease?).
14. Reports Summarize Gene Therapy Study Results from All India Institute of Medical Sciences (AIIMS) (Ocular perspective of mucopolysaccharidosis).
15. Researchers from University of Gdansk Publish Findings in Mucopolysaccharidoses (Mucopolysaccharidosis Type IIIE: A Real Human Disease or a Diagnostic Pitfall?).
16. Phase 1 Study of GC1130A in Pediatric Patients With Sanfilippo Syndrome Type A (MPS IIIA).
17. New Mucopolysaccharidoses Study Findings Recently Were Published by Researchers at Hospital Universitario Ramon y Cajal (The Role of the Gut Microbiota in Sanfilippo Syndrome's Physiopathology: An Approach in Two Affected Siblings).
18. New Mucopolysaccharidosis II Data Have Been Reported by Investigators at University of North Carolina (Initiation of Fluoxetine In a Pediatric Patient With Mucopolysaccharidosis Iiia: Early Observations).
19. New Mucopolysaccharidoses Findings from Centro Hospitalar Universitario de Sao Joao Described (Novel Fundoscopic Features in Mucopolysaccharidosis Type VI: Multimodal Evaluation of Scleral Deposits).
20. Patent Application Titled "Pharmaceutical Composition for Treatment of Mucopolysaccharidosis Type 1" Published Online (USPTO 20240269244).
21. Data on Mucopolysaccharidoses Detailed by a Researcher at SMS Medical College (Ventriculoperitoneal shunt and bilateral herniotomies in mucopolysaccharidosis type I: A surgical challenge).
22. Research from Harvard Medical School Provides New Data on Mucopolysaccharidoses (Intravenous Idursulfase for the Treatment of Mucopolysaccharidosis Type II: A Systematic Literature Review).
23. Investigators at University of North Carolina Chapel Hill Report Findings in Mucopolysaccharidoses (Community Consensus for Heparan Sulfate As a Biomarker To Support Accelerated Approval In Neuronopathic Mucopolysaccharidoses).
24. Findings from Pontificia University Javeriana Yields New Data on Mucopolysaccharidoses (Identification of Orthosteric and Allosteric Pharmacological Chaperones for Mucopolysaccharidosis Type Iiib).
25. Farwaniya Hospital Researchers Provide New Study Findings on Mucopolysaccharidoses (Enzymatic testing for mucopolysaccharidosis type I in Kuwaiti newborns: a preliminary study toward newborn screening).
26. An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB.
27. Research on Mucopolysaccharidoses Reported by Researchers at Amgen Research (Structural and mechanistic insights into a lysosomal membrane enzyme HGSNAT involved in Sanfilippo syndrome).
28. Department of Pediatric and Preventive Dentistry Reports Findings in Mucopolysaccharidoses (Caries assessment and salivary microbial analysis in patients diagnosed with mucopolysaccharidosis).
29. Researcher at Department of Pediatric Cardiology Publishes Research in Mucopolysaccharidoses [Evaluation of aortic elasticity properties in mucopolysaccharidosis patients; effect of enzyme replacement therapy (ERT) on aortic stiffness].
30. Researchers from Harbor-UCLA Medical Center Detail Findings in Mucopolysaccharidoses (Anakinra In Sanfilippo Syndrome: a Phase 1/2 Trial).
31. Landmark Nature Medicine study reports promising new treatment reduces suffering in Sanfilippo syndrome.
32. Extracellular vesicles from mucopolysaccharidosis type III microglia impair neurite growth.
33. Patent Issued for Treatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS) (USPTO 11986515).
34. New Mucopolysaccharidoses Study Findings Have Been Published by a Researcher at University of Uppsala (Inhibitors of dermatan sulfate epimerase 1 decreased accumulation of glycosaminoglycans in mucopolysaccharidosis type I fibroblasts).
35. National Taiwan University Hospital and School of Medicine Researchers Provide New Study Findings on Mucopolysaccharidoses (Mature neurons from iPSCs unveil neurodegeneration-related pathways in mucopolysaccharidosis type II: GSK-3b inhibition...).
36. Secondary deficiency of neuraminidase 1 contributes to CNS pathology in neurological mucopolysaccharidoses via hypersialylation of brain glycoproteins.
37. Children's Hospital of Zhejiang University School of Medicine Reports Findings in Mucopolysaccharidoses (Application of tandem mass spectrometry in the screening and diagnosis of mucopolysaccharidoses).
38. Cardiovascular damage in parents of patients with mucopolysaccharidoses.
39. Washington University School of Medicine Researchers Provide New Insights into Gene Therapy (Characterization of early markers of disease in the mouse model of mucopolysaccharidosis IIIB).
40. Takeda Development Center Americas Inc. Researchers Describe Advances in Mucopolysaccharidosis II (Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT...).
41. Research from National Institute of Health Yields New Study Findings on Mucopolysaccharidoses (Modeling Lysosomal Storage Disorders in an Innovative Way: Establishment and Characterization of Stem Cell Lines from Human Exfoliated Deciduous...).
42. Findings from University of Gdansk Provide New Insights into Mucopolysaccharidoses (Cellular Organelle-Related Transcriptomic Profile Abnormalities in Neuronopathic Types of Mucopolysaccharidosis: A Comparison with Other Neurodegenerative...).
43. Research from University of Minnesota Has Provided New Study Findings on Mucopolysaccharidoses (Natural history of craniovertebral abnormalities in a single-center study in 54 patients with Hurler syndrome).
44. New Mucopolysaccharidosis II Study Findings Recently Were Reported by Researchers at Takeda Development Center Americas Inc. (Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric...).
45. New Findings in Mucopolysaccharidosis II Described from Kumamoto University (Analysis of caregiver perspectives on patients with mucopolysaccharidosis II treated with pabinafusp alfa: results of qualitative interviews in Japan).
46. University Institute of Clinical Chemistry Researchers Provide Details of New Studies and Findings in the Area of Mucopolysaccharidoses (Specific GAG ratios in the diagnosis of mucopolysaccharidoses).
47. Pontifical University Javeriana Researchers Describe Recent Advances in Mucopolysaccharidoses (Evidence of epigenetic landscape shifts in mucopolysaccharidosis IIIB and IVA).
48. Liverpool Heart and Chest Hospital Researcher Updates Understanding of Mucopolysaccharidoses (Airway and Anaesthetic Management of Adult Patients with Mucopolysaccharidoses Undergoing Cardiac Surgery).
49. Ajou University Hospital Researchers Have Published New Study Findings on Mucopolysaccharidoses [Biomarkers of Glycosaminoglycans (GAG) accumulation in patients with mucopolysaccharidosis type VI-LeukoGAG, Corneal Opacification (COM) and Carotid...].
50. Data on Mucopolysaccharidoses Described by Researchers at Federal University Rio Grande do Sul (Effect of Genistein and Coenzyme Q10 In Oxidative Damage and Mitochondrial Membrane Potential In an Attenuated Type Ii Mucopolysaccharidosis...).
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