BioMarin to Present Research Results on Duchenne Muscular Dystrophy and Pompe Disease Programs at the 20th International Cong...
September 30 2015 - 4:06PM
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today that the
company will present research results at the 20th International
Congress of the World Muscle Society, including one oral
presentation in Pompe disease and two posters each in Duchenne
muscular dystrophy and Pompe disease programs. The meeting will be
held in Brighton, England from September 30 to October 4.
During an oral session, preliminary clinical efficacy and safety
data from an existing extension study of reveglucosidase alfa (BMN
701) in patients with late onset Pompe disease will be presented.
Pompe disease is an inherited condition caused by the deficiency in
the enzyme acid alpha-glucosidase, which leads to glycogen
accumulation and progressive muscle weakening, including muscles
essential for breathing. Reveglucosidase alfa is a novel fusion
protein of insulin-like growth factor 2 and acid alpha-glucosidase,
designed to target delivery to cell structures called lysosomes
where the enzyme is most needed.
Results will also be presented on Duchenne muscular dystrophy,
including those from a prospective natural history study to measure
progression of physical impairment, activity limitation and quality
of life in the condition. Duchenne muscular dystrophy is a
progressive muscle disorder caused by mutations in dystrophin, a
protein which plays an important structural role in muscle cells.
Boys living with Duchenne experience progressive muscle weakness,
resulting in serious medical complications and death.
A complete listing of the company's data presentations are as
follows:
Oral Presentation |
|
|
|
Title |
Authors |
G.O. 20: Reveglucosidase alfa (BMN 701), a
GILT-tagged recombinant human acid alpha glucosidase (rhGAA),
evaluation in late onset Pompe disease: Preliminary clinical
efficacy and safety results of an extension study (72-week
results) |
Roberts M, Hiwot T, Barohn R, Bratkovic D,
Byrne B, Desnuelle C, Hughes D, Laforet P, Mengel E, Yang K,
Heusner C, Dummer W |
|
|
Presentation: Sun.,
Oct. 4, 2015 from 8:45 - 9:00 |
|
|
|
Poster Presentations |
|
|
|
Pompe
disease |
|
Title |
Authors |
G.P. 8: An international, phase 3, switchover
study of reveglucosidase alfa (BMN 701) in subjects with late onset
Pompe disease (INSPIRE study) |
Schoser B, Byrne B, Eyskens F, Hiwot T,
Hughes D, Kissel J, Mengel E, Mozaffar T, Pestronk A, Roberts M,
Sivakumar K, Statland J, Young P, Heusner C, Dummer W |
|
|
Guided Poster
Session: Thurs., Oct. 1 from 14:30 - 16:00 |
|
G.P. 14: Lung function tests (MIP, MEP, VC,
FVC) predict ventilation and wheelchair use in late-onset Pompe
disease |
Roberts M, Mozaffar T, Young P, Johnson EM,
Quartel A, Berger KI. |
|
|
Guided Poster
Session: Thurs., Oct. 1 from 14:30 - 16:00 |
|
|
|
Duchenne Muscular
Dystrophy |
|
Title |
Authors |
G.P. 351: Exploring the route from exon
skipping antisense oligonucleotide administration to functional
effects in muscle of the mdx mouse model of Duchenne muscular
dystrophy |
Datson N, Weij R, Bijl S, Janson A, Vermue R,
Testerink J, van Deutekom J |
|
|
Guided Poster
Session: Fri., Oct. 2 from 16:30 - 18:00 |
|
G.P. 389: A prospective natural history study
to measure progression of physical impairment, activity limitation
and quality of life in Duchenne muscular dystrophy |
Goemans N, Wong B, McDonald C, Mason C, Hall
A, Campion G |
|
|
Guided Poster
Session: Fri., Oct. 2 from 16:30 - 18:00 |
|
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for patients with serious and
life-threatening rare and ultra-rare genetic diseases. The
company's portfolio consists of five commercialized products and
multiple clinical and pre-clinical product candidates. For
additional information, please visit www.BMRN.com.
CONTACT: Investors:
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media:
Debra Charlesworth
BioMarin Pharmaceutical Inc.
(415) 455-7451
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