Shire plc 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. Wednesday, February 7 from 11:45 am-12:45 pm PT. Seaport D-H (General Session Room)
SOURCE: Shire
Filed Under: Drug Discovery
