Latest news with #Mucopolysaccharidosis
Globe and Mail
20-05-2025
- Business
- Globe and Mail
Mucopolysaccharidosis Type I Pipeline Appears Robust With 8+ Key Pharma Companies Actively Working in the Therapeutics Segment
DelveInsight's, 'Mucopolysaccharidosis Type I Pipeline Insight 2025' report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Mucopolysaccharidosis Type I pipeline landscape. It covers the Mucopolysaccharidosis Type I pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Mucopolysaccharidosis Type I pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. Discover the latest drugs and treatment options in the Mucopolysaccharidosis Type I Pipeline. Dive into DelveInsight's comprehensive report today! @ Mucopolysaccharidosis Type I Pipeline Outlook Key Takeaways from the Mucopolysaccharidosis Type I Pipeline Report In May 2025, JCR Pharmaceuticals Co. Ltd announced a JR-171-101 study (NCT04227600) and fulfill all eligibility criteria can be enrolled in this JR-171-102 study. In the JR-171-102 study, subjects will receive either 2.0 or 4.0 mg/kg/week of JR-171 intravenously at the same doses received at Week 12 of the JR-171-101 study. DelveInsight's Mucopolysaccharidosis Type I Pipeline report depicts a robust space with 8+ active players working to develop 8+ pipeline therapies for Mucopolysaccharidosis Type I treatment. The leading Mucopolysaccharidosis Type I Companies such as Orchard Therapeutics, JCR Pharmaceuticals, REGENXBIO and others. Promising Mucopolysaccharidosis Type I Pipeline Therapies such as Laronidase, Antihistamine, Antipyretic, JR-171, AGT-181, Cyclosporine A (CsA), Azathioprine (Aza), RGX-111, SBC-103, and others. Stay ahead with the most recent pipeline outlook for Mucopolysaccharidosis Type I. Get insights into clinical trials, emerging therapies, and leading companies with DelveInsight @ Mucopolysaccharidosis Type I Treatment Drugs Mucopolysaccharidosis Type I Emerging Drugs Profile OTL 203: Orchard Therapeutics OTL-203 is an investigational ex vivo autologous gene therapy being investigated for the treatment of MPS-I. It uses a modified virus to insert a functional copy of the IDUA gene into a patient's cells. OTL-203 is being developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. OTL-203 has received rare pediatric disease designation from the FDA. Through an ongoing proof-of-concept clinical trial, OTL-203 is being evaluated as a potential treatment for patients with the most severe form of MPS-I, known as Hurler syndrome. OTL-203 is an investigational therapy and has not been approved by any regulatory agency or health authority. Currently, the drug is in Phase III stage of its development for the treatment of Mucopolysaccharidosis Type I. JR 171: JCR Pharmaceuticals JR-171, is an investigational drug for the treatment of mucoplysaccharidosis type I (MPS I, or Hurler, Hurler-Scheie and Scheie syndrome). JR-171 is a blood-brain-barrier (BBB)-penetrating form recombinant α-L-iduronidase that was developed using JCR's proprietary J-Brain Cargo® BBB technology. JR-171 received the orphan drug designation from the US Food Drug Administration (FDA) in February 2021. The trial is also scheduled for enrolling patients in the US. Currently, the drug is in Phase I/II stage of its development for the treatment of Mucopolysaccharidosis Type I. The Mucopolysaccharidosis Type I Pipeline Report Provides Insights into The report provides detailed insights about companies that are developing therapies for the treatment of Mucopolysaccharidosis Type I with aggregate therapies developed by each company for the same. It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Mucopolysaccharidosis Type I Treatment. Mucopolysaccharidosis Type I Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects. Mucopolysaccharidosis Type I Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Mucopolysaccharidosis Type I market Explore groundbreaking therapies and clinical trials in the Mucopolysaccharidosis Type I Marketed and Pipeline Drugs. Access DelveInsight's detailed report now! @ New Mucopolysaccharidosis Type I Drugs Mucopolysaccharidosis Type I Companies Orchard Therapeutics, JCR Pharmaceuticals, REGENXBIO and others. Mucopolysaccharidosis type I (MPS I) pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as Oral Intravenous Subcutaneous Parenteral Topical Mucopolysaccharidosis Type I Products have been categorized under various Molecule types such as Recombinant fusion proteins Small molecule Monoclonal antibody Peptide Polymer Gene therapy Unveil the future of Mucopolysaccharidosis Type I Treatment. Learn about new drugs, pipeline developments, and key companies with DelveInsight's expert analysis @ Mucopolysaccharidosis Type I Market Drivers and Barriers Scope of the Mucopolysaccharidosis Type I Market Report Coverage- Global Mucopolysaccharidosis Type I Companies- Orchard Therapeutics, JCR Pharmaceuticals, REGENXBIO and others. Mucopolysaccharidosis Type I Pipeline Therapies- Laronidase, Antihistamine, Antipyretic, JR-171, AGT-181, Cyclosporine A (CsA), Azathioprine (Aza), RGX-111, SBC-103, and others. Mucopolysaccharidosis Type I Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination Mucopolysaccharidosis Type I Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Get the latest on Mucopolysaccharidosis Type I Therapies and clinical trials. Download DelveInsight's in-depth pipeline report today! @ Mucopolysaccharidosis Type I Companies, Key Products and Unmet Needs Table of Contents Introduction Executive Summary Mucopolysaccharidosis type I (MPS I): Overview Pipeline Therapeutics Therapeutic Assessment Mucopolysaccharidosis type I (MPS I)– DelveInsight's Analytical Perspective Late Stage Products (Phase III) OTL 203: Orchard Therapeutics Drug profiles in the detailed report….. Mid Stage Products (Phase II) Drug Name: Company Name Drug profiles in the detailed report….. Early Stage Products (Phase I) JR 171: JCR Pharmaceuticals Drug profiles in the detailed report….. Preclinical and Discovery Stage Products Drug name : Company name Drug profiles in the detailed report….. Inactive Products Mucopolysaccharidosis type I (MPS I) Key Companies Mucopolysaccharidosis type I (MPS I) Key Products Mucopolysaccharidosis type I (MPS I)- Unmet Needs Mucopolysaccharidosis type I (MPS I)- Market Drivers and Barriers Mucopolysaccharidosis type I (MPS I)- Future Perspectives and Conclusion Mucopolysaccharidosis type I (MPS I) Analyst Views Mucopolysaccharidosis type I (MPS I) Key Companies Appendix About Us DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Yash Bhardwaj Email: Send Email Phone: 09650213330 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: NV Country: United States Website:
ITV News
07-05-2025
- Health
- ITV News
Scunthorpe mum discovers child had Hurler Syndrome after Googling 'bushy eyebrows'
Video report by Amelia Beckett. A mother who discovered her daughter had a rare genetic disorder after googling "bushy eyebrows" is calling for routine screening for the condition. Pamela Ives says she began searching online for facial features similar to those of her daughter, Sophia, after she began to lose physical similarities with her and her husband. She said: "From around six months, I knew there was something not right with her because she wasn't able to do tummy time, she had low muscle tone, and she was sensitive to light. "I happened to Google bushy eyebrows and then MPS (Mucopolysaccharidosis) came up. "[Google] said there's a one in 100,000 chance of your child having this syndrome. But I just knew that she had it from that point. "When I told my husband he thought I was going crazy. He said the condition is so so rare and there's no chance our daughter could have that." What is Hurler Syndrome? Mucopolysaccharidosis (MPS 1), also known as Hurler Syndrome, is a rare disorder where the body lacks an enzyme needed to break down complex sugar molecules. It causes the molecules to accumulate and when there's too much toxic buildup in cells they die or don't function properly. Children with Hurler Syndrome are usually not born with signs but develop symptoms during the first year of life. Symptoms include an enlarged head with prominent frontal bones, protruding eyes, enlarged lips and wide-open eyes. Children with this condition can also have heart valve problems, hearing loss, vision problems and respiratory issues. If Hurler Syndrome is not treated early it can be fatal. Developmental delay can become apparent by the age of 1 to 2 years and children with the condition have an average life expectancy of 5 years. Pamela added: "It wasn't until 14 months that we actually got the official diagnosis. "Personally I feel angry that it took until 14 months for her to get her diagnosis of MPS. "I want to raise awareness and I'm quite passionate about the UK changing it so they are tested on the new born screening. "I do know that some countries do test on the new born screening but unfortunately the UK isn't one of them." The Government previously said it does not recommend newborn screening for the condition. Treatment for Hurler Syndrome usually involves a bone marrow transplant before the age of two, for which a match is needed. But Sophia has been selected as one of 40 children worldwide for an experimental gene replacement therapy trial. It involves her own genes being sent to Milan where they will be modified and sent back to the UK to be implanted back into Sophia's body at Manchester Children's Hospital. Prof Robert Wynn: "Because she is her own donor it's actually a much safer transplant than using another person where we have to use drugs that immunosuppress and stop complications. "What I'm seeing after 25 years of looking after kids with hurler syndrome having transplants is a transformation of results." Sophia will also have to undergo chemotherapy which is required to weaken her immune system significantly.
