X
Pharma Advancement
  • Home
  • Drug Development
    • All
    • Clinical Trials
    • FDA Approvals
    • Research & Development
    Castor Launches CRO Partnership Program to Increase Adoption of Decentralized Clinical Trials

    Castor Launches CRO Partnership Program to Increase Adoption of Decentralized Clinical Trials

    Catalent acquires cell and gene therapy-focused MaSTherCell for $315m

    Cytiva and NecstGen form strategic collaboration to accelerate development of cell and gene therapies

    Astrazeneca Says Covid-19 Long-Acting Antibody Combination AZD7442 Rapidly Advances Into Phase III Clinical Trials

    Valneva and Pfizer Report Further Positive Phase 2 Data for Lyme Disease Vaccine Candidate

    BioMed X Institute and Merck KGaA, Darmstadt, Germany, Extend Collaboration in Oncology

    BioMed X Institute and Merck KGaA, Darmstadt, Germany, Extend Collaboration in Oncology

    New Combination Product for Symptoms of Seasonal Allergic Rhinitis Approved by US FDA with Aptar Pharma's VP3 Nasal Spray System

    New Combination Product for Symptoms of Seasonal Allergic Rhinitis Approved by US FDA with Aptar Pharma’s VP3 Nasal Spray System

    Cryoport and Cell Matters Enter into Strategic Partnership

    Cryoport and Cell Matters Enter into Strategic Partnership

    Evotec enters iPSC-based drug discovery partnership with Boehringer Ingelheim in ophthalmology

    Evotec enters iPSC-based drug discovery partnership with Boehringer Ingelheim in ophthalmology

    Gilead Announces Results From Phase 3 Trial of Remdesivir in Patients With Moderate COVID-19

    Gilead Sciences Announces FDA Approval of Veklury for Treatment of Non-Hospitalized Patients at High Risk for COVID-19

    Novavax to Participate in University of Oxford Com-COV2 Study Comparing Mixed COVID-19 Vaccine Combinations

    Australia Therapeutic Goods Administration Grants Provisional Registration for Novavax COVID-19 Vaccine

  • Manufacturing
  • Supply Chain
  • Facilities
  • Health & Nutrition
  • Events
  • Contact Us
No Result
View All Result
  • Home
  • Drug Development
    • All
    • Clinical Trials
    • FDA Approvals
    • Research & Development
    Castor Launches CRO Partnership Program to Increase Adoption of Decentralized Clinical Trials

    Castor Launches CRO Partnership Program to Increase Adoption of Decentralized Clinical Trials

    Catalent acquires cell and gene therapy-focused MaSTherCell for $315m

    Cytiva and NecstGen form strategic collaboration to accelerate development of cell and gene therapies

    Astrazeneca Says Covid-19 Long-Acting Antibody Combination AZD7442 Rapidly Advances Into Phase III Clinical Trials

    Valneva and Pfizer Report Further Positive Phase 2 Data for Lyme Disease Vaccine Candidate

    BioMed X Institute and Merck KGaA, Darmstadt, Germany, Extend Collaboration in Oncology

    BioMed X Institute and Merck KGaA, Darmstadt, Germany, Extend Collaboration in Oncology

    New Combination Product for Symptoms of Seasonal Allergic Rhinitis Approved by US FDA with Aptar Pharma's VP3 Nasal Spray System

    New Combination Product for Symptoms of Seasonal Allergic Rhinitis Approved by US FDA with Aptar Pharma’s VP3 Nasal Spray System

    Cryoport and Cell Matters Enter into Strategic Partnership

    Cryoport and Cell Matters Enter into Strategic Partnership

    Evotec enters iPSC-based drug discovery partnership with Boehringer Ingelheim in ophthalmology

    Evotec enters iPSC-based drug discovery partnership with Boehringer Ingelheim in ophthalmology

    Gilead Announces Results From Phase 3 Trial of Remdesivir in Patients With Moderate COVID-19

    Gilead Sciences Announces FDA Approval of Veklury for Treatment of Non-Hospitalized Patients at High Risk for COVID-19

    Novavax to Participate in University of Oxford Com-COV2 Study Comparing Mixed COVID-19 Vaccine Combinations

    Australia Therapeutic Goods Administration Grants Provisional Registration for Novavax COVID-19 Vaccine

  • Manufacturing
  • Supply Chain
  • Facilities
  • Health & Nutrition
  • Events
  • Contact Us
No Result
View All Result
Pharma Advancement
No Result
View All Result
Home Drug Development Clinical Trials

Treatment and Management of Rare Lysosomal Diseases highlighted by shire at Global Conference

Content Team by Content Team
8th February 2018
in Clinical Trials, Press Statements

Shire plc  the global biotechnology leader in rare diseases, today announced that it will have 12 presentations, including 11 posters and one oral presentation, at the 14th annual WORLDSymposium™ 2018 in San Diego, Calif., February 5-9. Shire will share research data for four lysosomal disease areas, including Hunter syndrome (also known as Mucopolysaccharidosis type II or MPS II), type 1 Gaucher disease, Fabry disease and metachromatic leukodystrophy (also known as MLD).

The company will also sponsor three satellite symposia and host an exhibit at WORLDSymposium™ (booth #301).
“We are proud to participate again at this year’s WORLDSymposium, a unique conference that focuses exclusively on lysosomal diseases, an area that formed the beginning of Shire’s strong commitment and focus on rare diseases,” said Hartmann Wellhoefer, M.D., Vice President, Head of Rare Diseases and Internal Medicine, Global
Medical Affairs, Shire. “This meeting brings together the world’s leading experts in the rare disease community to exchange the latest scientific knowledge and data about lysosomal diseases, along with advocacy groups who share valuable patient insights and perspectives. We look forward to sharing our data and contributing to the dialogue regarding earlier diagnosis, treatment and management of rare diseases.”

The congress is focused on sharing the latest information for lysosomal diseases, a collection of some 50 clinical syndromes, each resulting from the deficiency of a particular protein and causing a significant amount of disability and disease burden1.

Shire’s presence at the meeting includes the following key presentations, which are intended for scientific discussion only:

  • Infusion-related reactions in patients with mucopolysaccharidosis type II on idursulfase enrolled in the Hunter Outcome Survey (HOS): an update
    Poster #37, Tuesday, February 6, 2018 from 4:30-6:30 pm PT, Harbor Ballroom
  • Intrathecal delivery of recombinant human arylsulfatase A in children with late-infantile metachromatic leukodystrophy: a post hoc analysis of responders and non-responders
    Poster #118, Tuesday, February 6, 2018 from 4:30-6:30 pm PT, Harbor Ballroom
  • Metachromatic leukodystrophy and caregiver perspectives: understanding the natural history of the disease from interviews with caregivers
    Poster #135, Tuesday, February 6, 2018 from 4:30-6:30 pm PT, Harbor Ballroom
  • Menarche, menopause and pregnancy data in untreated females and females treated with agalsidase alfa in the Fabry Outcome Survey
    Poster #155, Tuesday, February 6, 2018 from 4:30-6:30 pm PT, Harbor Ballroom
  • Profile of natural history in 104 patients with mucopolysaccharidosis type II: insights from the Hunter Outcome Survey (HOS)
    Poster #172, Tuesday, February 6, 2018 from 4:30-6:30 pm PT, Harbor Ballroom
  • Management goals and normalization concept for type 1 Gaucher disease: results from a survey of expert physicians
    Poster #237, Wednesday, February 7, 2018 from 4:30-6:30 pm PT, Harbor Ballroom
  • Modeling and simulation of SHP611 in patients with metachromatic leukodystrophy (MLD): a preliminary evaluation
    Poster #380, Wednesday, February 7, 2018 from 4:30-6:30 pm PT, Harbor Ballroom
  • Projected Retained Ability Score (PRAS): a new methodology applied to DAS-II GCA scores for the longitudinal assessment of cognitive abilities in pediatric and adolescent patients with Hunter syndrome
    Poster #411, Wednesday, February 7, 2018 from 4:30-6:30 pm PT, Harbor Ballroom
  • Spectrum of genetic mutations in patients in the Fabry Outcome Survey (FOS)
    Poster #LB-19, Wednesday, February 7, 2018 from 4:30-6:30 pm PT, Harbor Ballroom
  • Demographic characteristics and GLA mutations in Taiwanese patients with Fabry disease: an analysis of data from the Fabry Outcome Survey (FOS)
    Poster #LB-24, Wednesday, February 7, 2018 from 4:30-6:30 pm PT, Harbor Ballroom
  • Long-term data analysis of 65 patients with Gaucher disease from the velaglucerase alfa clinical trial program who entered GOS
    Poster #LB-31, Wednesday, February 7, 2018 from 4:30-6:30 pm PT, Harbor Ballroom
  • Efficacy and safety of intrathecal idursulfase in pediatric patients with mucopolysaccharidosis type II and early cognitive impairment: design and methods of a controlled, randomized phase II/III multicenter study
    Oral presentation, Thursday, February 8, 2018 at 10:15 am PT, Seaport Ballroom
    Shire will also be sponsoring several symposia at the meeting:
    Sponsored by Shire Commercial
  • More Than a Glycolipid Storage Disorder – The Role of Inflammation in Fabry Disease
    Wednesday, February 7 from 6:30-7:30 am PT
    Seaport D-H (General Session Room)
    For Non-US Health Care Professionals only
    Sponsored by Shire Medical
  •  Normalization in Gaucher Disease – Does It Matter?
    Tuesday, February 6 from 6:30-7:30 am PT
    Seaport A-C
    For Non-US Health Care Professionals only
  • Cognitive Impairment in Patients with MPS II: From Disease Burden to Cognitive Testing

About the Genetic Diseases Franchise
Shire is a dedicated champion for people with rare diseases, which are typically inherited illnesses. Shire’s Genetic Diseases franchise has a strong legacy in developing therapies for lysosomal diseases, with a portfolio that includes commercial products and a robust R&D program with late-stage investigational therapies and pipeline candidates. The company’s commercial products address the following three disease areas:

  • Fabry Disease
    Fabry disease is a lysosomal disease affecting both males and females that interferes with the body’s ability to break down a specific fatty substance (globotriaosylceramide or Gb3) which accumulates within the body due to deficiency of a specific enzyme (α-galactosidase A)2. Fabry disease affects an estimated 1 in 117,000 live births3.
  • Hunter Syndrome
    Hunter syndrome is a severely debilitating, rare lysosomal disease caused by a deficiency of iduronate-2-sulfatase, an enzyme that is needed to break down substances in the body called glycosaminoglycans (GAGs)4. Without this enzyme, GAGs can build up, causing a range of disease-related signs and symptoms.4,5 Roughly two of every three patients with Hunter syndrome are also affected with progressive cognitive decline6. Hunter syndrome affects 1 in 162,000 total live births, and almost exclusively males.3
  • Type 1 Gaucher Disease
    Type 1 Gaucher disease is a rare, inherited metabolic condition, and the most common lysosomal disease. It affects approximately 1 in 100,000 people in the general population, and 1 in 855 people in the Ashkenazi Jewish community.7Patients with type 1 Gaucher disease may experience varying symptoms and degrees of disease severity, making type 1 Gaucher disease difficult to diagnose.8

For further information please contact:

Investor Relations
Christoph Brackmann christoph.brackmann@shire.com
+41 795 432 359

Sun Kim sun.kim@shire.com
+1 617 588 8175

Robert Coates rcoates@shire.com
+44 203 549 0874

Media

Annabel Cowper annabel.cowper@shire.com
+41 79 630 8619
Gwen Fisher gfisher@shire.com
+1 215 482 9649

Previous Post

Positive Results from the Pivotal Phase III ‘OPTIMISMM' Study of POMALYST/IMNOVID

Next Post

Shire presentations at EAHAD 2018 demonstrate commitment to studying outcomes in patients with bleeding disorders

Related Posts

Envirotainer introduces the RAP for the future - part of the Releye series
Packaging & Logistic

Envirotainer introduces the RAP for the future – part of the Releye series

14th February 2022
United Cargo Meets Growing Demand for Pharmaceuticals with New TempControl Containers
Packaging & Logistic

Opticooler receives technical approval by Japanese major airlines

14th February 2022
Aptar Pharma Launches HeroTracker Sense, Novel Connected Smart Device for Respiratory Disease
Press Statements

Aptar Pharma Launches HeroTracker Sense, Novel Connected Smart Device for Respiratory Disease

14th February 2022
Castor Launches CRO Partnership Program to Increase Adoption of Decentralized Clinical Trials
Clinical Trials

Castor Launches CRO Partnership Program to Increase Adoption of Decentralized Clinical Trials

7th February 2022
Novavax to Participate in University of Oxford Com-COV2 Study Comparing Mixed COVID-19 Vaccine Combinations
Press Statements

New Zealand’s Medsafe Grants Provisional Approval for Novavax COVID-19 Vaccine

7th February 2022
First own Gerresheimer Autoinjector - Gerresheimer and Midas Pharma announce strategic partnership
Packaging & Logistic

The leading supplier of injection vials Gerresheimer expands production in Wertheim

7th February 2022
Next Post

Shire presentations at EAHAD 2018 demonstrate commitment to studying outcomes in patients with bleeding disorders

Latest News

Envirotainer introduces the RAP for the future - part of the Releye series
Packaging & Logistic

Envirotainer introduces the RAP for the future – part of the Releye series

14th February 2022
United Cargo Meets Growing Demand for Pharmaceuticals with New TempControl Containers
Packaging & Logistic

Opticooler receives technical approval by Japanese major airlines

14th February 2022
Aptar Pharma Launches HeroTracker Sense, Novel Connected Smart Device for Respiratory Disease
Press Statements

Aptar Pharma Launches HeroTracker Sense, Novel Connected Smart Device for Respiratory Disease

14th February 2022
Johnson & Johnson Prepares to Resume Phase 3 ENSEMBLE Trial of its Janssen COVID-19 Vaccine Candidate in the U.S.
News

Indian Scientists Claim To Design Vaccine For All Variants

9th February 2022
Drones for Delivery To Carry Pharmaceutical Weight Forward
News

Drones for Delivery To Carry Pharmaceutical Weight Forward

9th February 2022
Pharma Advancement

About Us

Pharma Advancement is a leading Pharma information centric website. On one side Pharmaadvancement.com has established itself as one of the most efficient and comprehensive source of Pharma information online, dedicated to providing decision makers in all the Pharma industry sectors with reliable, accurate and useful insights into happenings in the Pharma sector.

Qucik Links

  • Drug Development
  • Manufacturing
  • News
  • Events & Conferences
  • Newsletter Archive

Resources

  • Advertise with us
  • Contact Us
  • Download Mediapack

System

  • Search
  • Sitemap
  • RSS Feed

© 2017 Copyright © Valuemediaservices 2017 All rights reserved.

No Result
View All Result
  • Home
  • Drug Development
  • Manufacturing
  • Supply Chain
  • Facilities
  • Health & Nutrition
  • Events
  • Contact Us

© 2017 Copyright © Valuemediaservices 2017 All rights reserved.

Login to your account below

Forgotten Password?

Fill the forms bellow to register

All fields are required. Log In

Retrieve your password

Please enter your username or email address to reset your password.

Log In