Glycosaminoglycan

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

Retrieved on: 
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

FDA Clears Groundbreaking Corplex P™: The First Human Umbilical Cord-Derived Medical Device

Retrieved on: 
Tuesday, February 6, 2024
ECM, Patient, Safety, Collagen, Glycosaminoglycan, FDA, Medical device

ROSWELL, Ga., Feb. 6, 2024 /PRNewswire/ -- StimLabs® announces FDA 510(k) clearance of Corplex P, the pioneering human umbilical cord-derived medical device.

Key Points: 
  • ROSWELL, Ga., Feb. 6, 2024 /PRNewswire/ -- StimLabs® announces FDA 510(k) clearance of Corplex P, the pioneering human umbilical cord-derived medical device.
  • Corplex P is a particulate device derived from human umbilical cord extracellular matrix (ECM) indicated for the management of acute and chronic wounds.
  • This designates StimLabs as the first company ever to receive a wound management 510(k) clearance for human birth tissue products.
  • "The clearance of Corplex P changes the landscape for bringing human birth tissue-based products indicated for wound management to the market."

Orchard Therapeutics Announces First Patient Randomized in Registrational Trial of OTL-203 for MPS-I Hurler Syndrome

Retrieved on: 
Monday, February 5, 2024
Partnership, Mortality, Hurler, Orchard, Growth, Congress, Cell, Division, Heart, ERT, Glycosaminoglycan, GAG, Health, Disease, Patient, Safety, Transplant, FDA, EMA, Șaeș, San Raffaele Hospital, ESGCT, HSCT, Food, POC, Therapy, MPS, Hurler syndrome, Kyowa Kirin, Acquisition, Enzyme replacement therapy, European Medicines Agency, Gene, University, Eye, IDUA, HSC, RPD, Pharmaceutical industry

TOKYO, LONDON and BOSTON, Feb. 05, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the first patient has been randomized at the M Health Fairview Masonic Children’s Hospital in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic stem cell (HSC) gene therapy, in patients with the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial, referred to as HURCULES, compares treatment with OTL-203 to standard of care with allogeneic hematopoietic stem cell transplant (HSCT), and is expected to enroll 40 MPS-IH patients at sites across the U.S. and Europe.

Key Points: 
  • Approximately 60 percent of children born with MPS-I have the most severe subtype, MPS-IH, also called Hurler syndrome, and rarely live past the age of 10 when untreated.
  • Current treatment options for MPS-IH include allogeneic hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy (ERT), both of which have significant limitations.
  • One patient experienced an acute hypersensitivity reaction that was considered probably related to HSC gene therapy.
  • The viral vector integration profile was comparable with other Orchard Therapeutics lentiviral-based HSC gene therapy studies, and all participants had a stable and highly polyclonal repertoire.

Orchard Therapeutics Receives U.S. FDA Fast Track Designation for OTL-203 in MPS-IH

Retrieved on: 
Thursday, November 30, 2023
Quality of life, Glycosaminoglycan, Food, ESGCT, Health, Antibody, FDA, Cell, Disease, Mortality, Tissue, Congress, Gene, HSC, POC, Therapy, RPD, Heart, Hurler syndrome, Eye, San Raffaele Hospital, Death, San Raffaele, ERT, European Medicines Agency, Cognitive development, IDUA, Growth, HSCT, Patient, Safety, Diagnosis, Hurler, Fast Track, Pharmaceutical industry

BOSTON and LONDON, Nov. 30, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OTL-203, an investigational hematopoietic stem cell gene (HSC) therapy being developed for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). Fast Track designation is intended to expedite the development of promising medicines that address serious medical needs. Therapeutic candidates that receive Fast Track designation may be eligible for enhanced interactions with the FDA, including potentially quicker submission and review timelines.

Key Points: 
  • Fast Track designation is intended to expedite the development of promising medicines that address serious medical needs.
  • Therapeutic candidates that receive Fast Track designation may be eligible for enhanced interactions with the FDA, including potentially quicker submission and review timelines.
  • “We are encouraged that OTL-203 has been granted Fast Track designation because new treatment options are urgently needed for children with MPS-IH, as the current standard of care is associated with significant morbidity and mortality.
  • Throughout the PoC study, treatment with OTL-203 has been generally well-tolerated with a safety profile consistent with the selected conditioning regimen.

Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

Retrieved on: 
Monday, December 18, 2023
CNS, PPQ, Regenerative medicine, Brain, GMP, MPS, PRIME, CDMO, Sanfilippo syndrome, Regenerative Medicine Advanced Therapy, History, Patient, Research, SGSH, Cell damage, Process performance qualification protocol, EVP, Glycosaminoglycan, Partnership, Development, Orphan drug, Cell, AAV, Pharmaceutical industry, Ultragenyx

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).

Key Points: 
  • COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).
  • Andelyn has successfully manufactured GMP supply of UX111 for patient dosing and will be performing the PPQ batches necessary for any future regulatory filings.
  • Andelyn is honored to continue its close partnership to advance the UX111 Program to bring treatment to MPS IIIA patients sooner."
  • With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn is enabling the progression of life-altering gene therapies for rare and prevalent diseases.

Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

Retrieved on: 
Monday, December 18, 2023
CNS, PPQ, Regenerative medicine, Brain, GMP, MPS, PRIME, CDMO, Sanfilippo syndrome, Regenerative Medicine Advanced Therapy, History, Patient, Research, SGSH, Cell damage, Process performance qualification protocol, EVP, Glycosaminoglycan, Partnership, Development, Orphan drug, Cell, AAV, Pharmaceutical industry, Ultragenyx

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).

Key Points: 
  • COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).
  • Andelyn has successfully manufactured GMP supply of UX111 for patient dosing and will be performing the PPQ batches necessary for any future regulatory filings.
  • Andelyn is honored to continue its close partnership to advance the UX111 Program to bring treatment to MPS IIIA patients sooner."
  • With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn is enabling the progression of life-altering gene therapies for rare and prevalent diseases.

Orchard Therapeutics Announces Presentation of Additional Positive Data from Proof-of-concept Study of OTL-203 in MPS-IH at ESGCT 2023

Retrieved on: 
Thursday, October 26, 2023
Glycosaminoglycan, Heart, Hurler syndrome, Hearing loss, Congress, Photophobia, POC, ERT, IDUA, Cell, Disease, MPS, Antibody, Gene, San Raffaele Hospital, HSC, The New England Journal of Medicine, ESGCT, Eye, Safety, European Society for Primary Immunodeficiencies, Therapy, Patient, Diagnosis, Death, Hurler, San Raffaele, European Society of Gene and Cell Therapy, HSCT, Pharmaceutical industry, Medical device, Medical imaging

The data were presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27, 2023, in Brussels.

Key Points: 
  • The data were presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27, 2023, in Brussels.
  • “The complications associated with MPS-IH involve multiple organ systems and have an adverse impact on patients’ quality of life.
  • Importantly, following treatment with OTL-203, no patients reported photophobia (light sensitivity), or any other ophthalmological symptoms typically associated with MPS-IH.
  • Secondary endpoints include biochemical markers, additional clinical assessments, as well as safety and tolerability.

Can I put cortisone on my face? The right advice on creams to fix irritated skin

Retrieved on: 
Tuesday, June 20, 2023
Rosacea, Human, Skin, Vein, Atrophoderma, Education, Face, Solution, Dermatitis, Connective tissue, Telangiectasia, Finger, Cortisone, Acne, Research, Psoriasis, Capillary, Perioral dermatitis, Hydrocortisone, Erythema, Patient, Risk, Steroid, Vitiligo, Sunburn, Glycosaminoglycan, Groin, Maharashtra University of Health Sciences, News, Red, Sulfur, Vaccine, Dietary supplement, Mineral oil, Cosmetics, Topical medication

Topical corticosteroids, also referred to as corticosteroids or cortisone, are the go-to treatment for many skin disorders including eczema, dermatitis and psoriasis.

Key Points: 
  • Topical corticosteroids, also referred to as corticosteroids or cortisone, are the go-to treatment for many skin disorders including eczema, dermatitis and psoriasis.
  • They can be found in various forms such as creams, ointments, and solutions.
  • Topical corticosteroids come in different strengths, from mild (such as 0.5% Hydrocortisone, available over the counter) to very potent prescription formulations.

‘Use sparingly’

    • Such warnings ignore the fact many patients are prescribed modest-strength topical steroids, which are safe and effective when used properly.
    • From the patient’s perspective, the current warnings lump all steroids together regardless of their potential for side effects.

Mixed messages

    • Or hit the problem hard with a stronger concentration for less time?
    • If cortisone is extensively used, it is advised adults and children should be examined yearly for side effects.
    • Skin atrophy (or deterioration) is the most common side effect of topical corticosteroids and manifests as tiny degenerative alterations within a few weeks.

Skin thinning

    • Repeated use in the same spot on the body results in alterations to the skin’s connective tissue and epidermal thinning.
    • That can result in lax, translucent, wrinkled skin as well as striae (stretch marks), fragility, hypo-pigmentation (fading) and the prominence of underlying veins.
    • The good news is that once topical corticosteroids are stopped, short-term atrophy from treatment can be reversed, although skin normalisation may take months.

Can you use it safely on your face?

    • Using strong cortisone creams or ointments on the face can lead to steroid dependence.
    • Topical steroids on the face can cause symptoms sometimes referred to as “red face syndrome”, dermatitis rosaceaformis steroidica or steroid addiction.
    • And stopping steroid use on the face after an extended period can have considerable rebound effects including erythema (redness), burning and scaling.

The bottom line

    • To avoid skin damage, corticosteroids should only be used on skin affected by a skin disease.
    • Finally, treatment should be customised based on the person’s symptoms, the body parts affected and how long treatment might be required.
    • This article reflects the views of the author and
      should not be construed to represent views or
      policies.

YuMOVE Explains the Importance of Triple Action Joint Support for Dogs

Retrieved on: 
Thursday, June 1, 2023
Glucosamine, Radical, American Kennel Club, Manganese, Hip, Synovial fluid, Vitamin E, Ligament, Dog, Cartilage, Royal Veterinary College, Glycosaminoglycan, Tendon, Dietary supplement

ORLANDO, Fla., June 1, 2023 /PRNewswire/ -- YuMOVE , a high-quality joint supplement for dogs and the American Kennel Club's official dog joint supplement, offers triple-action formula to ease occasional stiffness, support joint structure and promote mobility in dogs.

Key Points: 
  • ORLANDO, Fla., June 1, 2023 /PRNewswire/ -- YuMOVE , a high-quality joint supplement for dogs and the American Kennel Club's official dog joint supplement, offers triple-action formula to ease occasional stiffness, support joint structure and promote mobility in dogs.
  • YuMOVE joint care is based on more than 15 years of scientific research.
  • Supports joint structure – YuMOVE contains glucosamine, the basic building blocks of cartilage helping support joint structure.
  • YuMOVE is offering 30% off any YuMOVE hip and joint supplement for dogs using the code "YuMOVEMENT" for a one-time purchase.

REGENXBIO Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RGX-121 Gene Therapy for Hunter Syndrome

Retrieved on: 
Tuesday, May 23, 2023
Drug development, Pediatrics, University, MPS II, University of North Carolina, Quality of life, Chapel Hill, Syndrome, MPS, AAV, Dementia, II, HS, Mucopolysaccharidosis type I, Genetics, Acquisition, University of California, San Francisco Fetal Treatment Center, Patient, Glycosaminoglycan, CSF, Phase, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Hunter syndrome, Food, Disease, 21st Century Cures Act, Medical device, Pharmaceutical industry

"I am encouraged by the FDA's recognition of the potential of a gene therapy like RGX-121 to improve the quality of life for boys with severe Hunter syndrome and the urgent need for new treatment options."

Key Points: 
  • "I am encouraged by the FDA's recognition of the potential of a gene therapy like RGX-121 to improve the quality of life for boys with severe Hunter syndrome and the urgent need for new treatment options."
  • "The expedited development of new treatment options like RGX-121 are critical to families facing this devastating diagnosis."
  • In addition, improvements in neurodevelopmental and daily activity skill acquisition were observed up to three years after RGX-121 administration.
  • A Phase I/II trial of RGX-121 for the treatment of pediatric patients with MPS II over the age of five years old is also ongoing.