CAMBRIDGE, Massachusetts, February 14, 2017 /PRNewswire/ --
14 presentations and 5 satellite symposia across 3 lysosomal storage disorders at important scientific meeting represent Shire's continued focus on the diagnosis, treatment, and management of these life-altering diseases
Shire plc (LSE: SHP, NASDAQ: SHPG), the global leader in rare diseases, will be presenting 14 posters, including two late-breaking abstracts, at the 13th annual WORLDSymposium™ 2017 in San Diego, Calif., (February 13-17). Presentations are focused on data in lysosomal storage diseases (LSDs), including Hunter syndrome (also known as Mucopolysaccharidosis type II or MPS II), type 1 Gaucher disease, and Fabry disease, and highlight Shire's commitment to innovative research in genetic diseases.
"We are proud of our ongoing commitment to patients with rare genetic diseases and look forward to showcasing the depth and breadth of our scientific research across the LSDs," said Hartmann Wellhoefer, M.D., Vice President, Head of Medical Affairs, Genetic Diseases. "The data we will be presenting represent our continued focus on advancing the diagnosis, treatment, and management of life-altering diseases."
Each year, WORLDSymposium hosts a scientific meeting presenting the latest information from basic science, translational research, and clinical trials for lysosomal diseases. This symposium is designed to educate researchers and clinicians to better understand diagnostic and management options for patients with lysosomal diseases; identify areas requiring additional basic and clinical research, public policy and regulatory attention; and present the latest findings in the natural history of lysosomal diseases.
At the meeting, in addition to data presentations, Shire also will be sponsoring three booths (#12, 16, and 19) in the exhibition hall, as well as five satellite symposia. Following are the key Shire presentations at the meeting:
- A long-term extension study evaluating intrathecal idursulfase-IT in children with Hunter Syndrome and cognitive impairment (HIT-045/046)
Poster #240, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- Clinical outcomes after 3 years of idursulfase treatment in patients with MPS II: data from the Hunter Outcome Survey (HOS)
Poster #114, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- The Challenge of using Hospital Episode Statistics to identify a Hunter Syndrome Cohort in the UK
Poster #231, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- Health care resource utilization by patients with Hunter Syndrome in the UK Hospital Episode Statistics database
Poster #230, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- Validation of the shortened Hunter Syndrome-functional outcomes for clinical understanding scale (HS-FOCUS) questionnaire
Poster #220, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- Profile of patients with Mucopolysaccharidosis type II without cognitive impairment who started idursulfase treatment aged >20 years: data on late treatment initiation from the Hunter Outcome Survey (HOS)
Poster #185, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- Characteristics of patients with Mucopolysaccharidosis type II diagnosed ≥5 years of age: data from the Hunter Outcome Survey (HOS)
Poster #134, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- Intrafamilial variability in the clinical presentation of Mucopolysaccharidosis type II: data from the Hunter Outcome Survey (HOS)
Poster #85, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- Urinary glycosaminoglycan levels in a Mucopolysaccharidosis type II pediatric population receiving idursulfase therapy: data from the Hunter Outcome Survey (HOS) for patients aged <18 months
Poster #8, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- Medical histories in Gaucher disease: a descriptive analysis from 852 patients in the Gaucher Outcome Survey (GOS)
Poster #82, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- Characteristics of 27 patients with type 3 Gaucher disease: a descriptive analysis from the Gaucher Outcome Survey
Poster #303, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- Real-world treatment patterns from 647 patients with Gaucher disease: an analysis from the Gaucher Outcome Survey
Poster #70, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- Outcomes of 453 pregnancies in patients with Gaucher disease: an analysis from the Gaucher Outcome Survey
Poster #184, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- Prompt agalsidase alfa therapy initiation after symptom onset is associated with improved renal and cardiovascular outcomes in the Fabry Outcome Survey
Poster #199, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- The Australian ATYOURSIDE patient support experience
Poster #LB-09, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
- The Australian ATHOME™ infusion service experience
Poster #LB-19, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom
Shire is dedicated to addressing challenges in the LSD community, and will be sponsoring several symposia at the meeting:
Sponsored by Shire Commercial
- On the Frontlines of Managing Patients with Lysosomal Diseases
Tues, February 14 from 6:30-7:30 am PT
General Session Room (Seaport Ballroom D-H)
- Pioneers and Innovators in Gaucher Disease
Wed, Feb 15 from 11:45 am-12:45 pm PT
General Session Room (Seaport Ballroom D-H)
Sponsored by Shire Medical
- MPS II: One Family's Journey
Tues, February 14 from 11:45 am - 12:45 pm PT
General Session Room (Seaport Ballroom D-H)
- Discovery and Research Platforms in Rare Disease
Tues, Feb 14 from 6:30-8:30 pm PT
General Session Room (Seaport Ballroom D-H)
- MPS II with Cognitive Impairment: Approaches to Improving Identification and Assessment
Wed, Feb 15 from 6:30-7:30 am PT
Seaport Ballroom A-C
About the Genetic Disease Franchise
Shire is dedicated to helping patients with inherited illnesses. Shire's genetic disease franchise has a strong legacy in developing therapies for LSDs, with a portfolio that includes commercial products, late-stage investigational therapies, and pipeline candidates, as well as a robust R&D program.
Hunter syndrome, or mucopolysaccharidosis II (MPS II), is a serious genetic disorder that primarily affects males. It interferes with the body's ability to break down and recycle specific mucopolysaccharides, also known as glycosaminoglycans or GAGs. Hunter syndrome is one of several related lysosomal storage diseases.
Type 1 Gaucher Disease
Type 1 Gaucher disease is a rare, inherited metabolic condition, and the most common of a family of rare diseases known as lysosomal storage disorders (LSDs). It affects approximately 1 in 100,000 people in the general population, and 1 in 855 people in the Ashkenazi Jewish community. Patients with type 1 Gaucher disease may experience varying symptoms and degrees of disease severity, making type 1 Gaucher disease difficult to diagnose.
Fabry disease is a lysosomal storage disorder (LSD) 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). Fabry disease affects an estimated 8,000 to 10,000 people worldwide.
NOTES TO EDITORS
Shire is the leading global biotechnology company focused on serving people with rare diseases and other highly specialized conditions. We strive to develop best-in-class products, many of which are available in more than 100 countries, across core therapeutic areas including Hematology, Immunology, Neuroscience, Ophthalmics, Lysosomal Storage Disorders, Gastrointestinal / Internal Medicine / Endocrine and Hereditary Angioedema; and a growing franchise in Oncology.
Our employees come to work every day with a shared mission: to develop and deliver breakthrough therapies for the hundreds of millions of people in the world affected by rare diseases and other high-need conditions, and who lack effective therapies to live their lives to the fullest.
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Marie von Seyfried
SOURCE Shire plc