Scheie syndrome

Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

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Thursday, April 18, 2024
Eurostat, Diagnosis, Civil service commission, Death, Glycosaminoglycan, B cell, Enzyme, Mutation, Medicine, Cell, Mental disability, Disease, Patient, Committee, Knowledge, Scheie syndrome, Regulation, EMA, IRIS, Periodic breathing, Mucopolysaccharidosis, European, COMP, Enzyme replacement therapy, European Commission, Safety, DSM-IV codes, IQVIA, Marketing, Haematopoiesis, Liver, RDS, FGK, Hurler syndrome, MPS, Pharmaceutical industry

Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

Key Points: 


Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positive

JCR Pharmaceuticals’ Research Presentations at WORLDSymposium™ 2024 Showcase JR-141 (Pabinafusp Alfa) and Other Investigational Treatments for Lysosomal Storage Disorders

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Wednesday, February 14, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Clinical Trials, Enzyme replacement therapy, BBB, JCR, MPS II, Traffic barrier, Glycogen storage disease, Ministry, MHLW, ERT, Hunter syndrome, Syndrome, Scheie syndrome, MPS, Safety, Developmental psychology, Hurler syndrome, Child, Patient, Hurler–Scheie syndrome, Trial of the century, Consciousness, DSM-IV codes, Hypotension, Mouse, Pharmaceutical industry

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).
  • JCR is dedicated to address the unmet medical needs for this community,” said Shin Ashida, President and CEO of JCR Pharmaceuticals.
  • Finally, the third JR-141 presentation is pre-clinical and highlights the recovery of retinal function in mice with MPS II.
  • “We are pleased to be able to offer IZCARGO® to patients with MPS II in Japan,” said Yoshikatsu Eto, M.D., Ph.D., Institute of Neurological Disorders, Advanced Clinical Research Center, Kanagawa, Japan.

JCR Pharmaceuticals to Present at the 20th Annual WORLDSymposium™ 2024

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Thursday, January 25, 2024
Health, Other Science, General Health, Research, Science, Pharmaceutical, Biotechnology, Hurler, Patient, Enzyme replacement therapy, PST, Hunter syndrome, MPS II, JCR, Eto, BBB, MPS, Medicine, MHLW, Ikeda, ERT, Syndrome, Safety, Traffic barrier, Glycogen storage disease, Scheie syndrome, Ministry, Pharmaceutical industry

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that it will present six presentations at the 20th Annual WORLDSymposium™ 2024, to be held February 4-9, 2024 in San Diego, Calif.

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that it will present six presentations at the 20th Annual WORLDSymposium™ 2024, to be held February 4-9, 2024 in San Diego, Calif.
  • In addition to posters being available for each of the presentations, two of them will be shared as oral presentations given by a clinical investigator and a researcher from JCR Pharmaceuticals.
  • A global phase III study of pabinafusp alfa (JR-141) for neuronopathic mucopolysaccharidosis type II: updated study design (Ikeda, et al.)
  • WORLDSymposium™ attendees who would like to receive more information about JCR Pharmaceuticals can visit JCR’s on-site conference booth (#506) or visit its virtual booth on the WORLDSymposium™ conference website.

EMA grants Orphan Drug Designation to JR-171 for the Treatment of Mucopolysaccaridosis Type I (MPS I)

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Tuesday, March 30, 2021
Research, Clinical trials, Biotechnology, Other Health, Health, Pharmaceutical, General Health, Other Science, Science, Health, Life sciences, Clinical research, Pharmaceuticals policy, Syndromes, Food and Drug Administration, Drug discovery, Orphan drug, Scheie syndrome, European Medicines Agency, Investigational New Drug, Hurler, MPS I (Hurler, Hurler-Scheie, Scheie syndrome), J-Brain Cargo® Technology, JCR Pharmaceuticals Co., Ltd.

30, 2021 -- JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; JCR) announced today that European Medicines Agency (EMA) has granted orphan drug designation to JR-171, an investigational drug for the treatment of mucoplysaccharidosis type I (MPS I, or Hurler, Hurler-Scheie and Scheie syndrome).

Key Points: 
  • 30, 2021 -- JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; JCR) announced today that European Medicines Agency (EMA) has granted orphan drug designation to JR-171, an investigational drug for the treatment of mucoplysaccharidosis type I (MPS I, or Hurler, Hurler-Scheie and Scheie syndrome).
  • JR-171 received the orphan drug designation from the US Food Drug Administration (FDA) in February 2021.
  • This designation is expected to have a minor impact on JCR's consolidated financial results for the year ending on March 31, 2021.
  • The EMA implements orphan designation for promoting new drug development for rare diseases in which the prevalence of the condition in the EU must not be more than 5 in 10,000.

Epiomic Epidemiology Report: Mucopolysaccharidosis Type Vi Forecast In 30 Major Markets 2018-2028

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Tuesday, July 17, 2018
Syndromes, Medicine, Maroteaux–Lamy syndrome, Mucopolysaccharidosis, Health, Genetic disorder, Clinical medicine, Scheie syndrome, Mucopolysaccharidosis type I

The "Epiomic Epidemiology Series: Mucopolysaccharidosis Type Vi Forecast In 30 Major Markets 2018-2028" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Epiomic Epidemiology Series: Mucopolysaccharidosis Type Vi Forecast In 30 Major Markets 2018-2028" report has been added to ResearchAndMarkets.com's offering.
  • Mucopolysaccharidosis type VI (MPS VI), also known as Maroteaux-Lamy syndrome, is an inherited autosomal recessive genetic disorder in which glycosaminoglycans (GAGs) are accumulated in lysosomes due to mutations that cause loss of function of an enzyme responsible for degrading GAGs.
  • MPS VI constitutes a burden to the patients and their families as the disease progresses and the patients develop mobility impairment, heart problems and corneal clouding.
  • These sub-populations within the main disease are also included at a country level across the 10-year forecast snapshot.