Long-term functional data from Sarepta Therapeutics’ Most Advanced Gene Therapy Programs to be Presented at Upcoming Annual...
14 September 2020 - 10:30PM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today announced that new data
from its most advanced gene therapy programs will be presented at
the WMS25 Virtual Congress, the 25th International Annual Congress
of the World Muscle Society, being held Sept. 28 – Oct. 2.
Sarepta will host a webcast and conference call
on Monday, Sept. 28, 2020 at 8:30 a.m. ET, to discuss the results,
which include two-year functional data from Study 101 of SRP-9001
for Duchenne muscular dystrophy and 18-month functional results
from Cohort 1 in the study of SRP-9003 for Limb-girdle muscular
dystrophy Type 2E.
This will be webcast live under the investor
relations section of Sarepta's website at
https://investorrelations.sarepta.com/events-presentations and
will be archived there following the call for one year. Please
connect to Sarepta's website several minutes prior to the start of
the broadcast to ensure adequate time for any software download
that may be necessary. The conference call may be accessed by
dialing (844) 534-7313 for domestic callers and (574) 990-1451 for
international callers. The passcode for the call is 6793650. Please
specify to the operator that you would like to join the "Long-term
Functional Data from Sarepta’s Gene Therapy Programs” call.
In total, Sarepta will present 16 abstracts at
this year’s meeting. All posters will be available on-demand
throughout the Congress beginning on Monday, Sept. 28 at 7:00 a.m.
EST. The full WMS25 Virtual Congress program is available here:
https://www.wms2020.com/programme/.
Gene Therapy:
TITLE |
PROGRAM |
POSTER # |
Treatment of Aged Mice and Long-term Durability of AAV-Mediated
Gene Therapy in Two Mouse Models of Limb Girdle Muscular
Dystrophy |
SRP-9003, SRP-9004 |
P.137 |
Expression-Functional Correlation and Validation of a Surrogate
Marker for DAPC Restoration in LGMD2E Mouse Model |
SRP-9003 |
P.139 |
Systemic Gene Transfer with rAAVrh74.MHCK7.SGCB Increased
β-sarcoglycan Expression in Patients with Limb Girdle Muscular
Dystrophy Type 2E |
SRP-9003 |
P.140 |
Evaluation of the Lipid-Binding and Stability Properties of
Recombinant Dystrophin Spectrin-Like Repeat Constructs |
SRP-9001 |
P.206 |
Systemic Gene Transfer with rAAVrh74.MHCK7.micro-dystrophin in
Patients with Duchenne Muscular Dystrophy |
SRP-9001 |
P.280 |
Systemic Dose-Finding Study with AAV-Mediated γ-Sarcoglycan Gene
Therapy for Treatment of Muscle Deficits in LGMD2C Mice |
SRP-9005 |
P.138 |
RNA Platform:
TITLE |
PROGRAM |
POSTER # |
Long-term Safety and Efficacy of Golodirsen in Male Patients with
Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping |
Golodirsen |
P.283 |
Casimersen Treatment in Eligible Patients with Duchenne Muscular
Dystrophy: Safety, Tolerability, and Pharmacokinetics Over 144
Weeks of Treatment |
Casimersen |
P.288 |
Open-Label Evaluation of Eteplirsen in Patients With Duchenne
Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial |
Eteplirsen |
P.289 |
Delay in Duchenne Muscular Dystrophy Progression with Eteplirsen:
Attenuation of Pulmonary Decline and Projected Freedom from
Continuous Ventilation |
Eteplirsen |
P.290 |
Real-world Evidence of Eteplirsen Treatment Effects on Duchenne
Muscular Dystrophy Related Health Outcomes Using Claims Data in the
United States |
Eteplirsen |
P.291 |
Natural history and other
presentations:
TITLE |
POSTER # |
Development of Cardiomyopathy, Respiratory Insufficiency and Loss
of Ambulation in Becker Muscular Dystrophy: A Systematic Literature
Review |
P.41 |
Disease Attributes Most Important from a Societal Perspective: A
Case Study Involving Duchenne Muscular Dystrophy |
P.56 |
Identification of Disease Progression Stages in Patients with
Duchenne Muscular Dystrophy Using Administrative Claims Data in the
United States |
P.119 |
The Age at Loss of Ambulation Among Patients with Limb-Girdle
Muscular Dystrophy (LGMD) Subtype 2: A Systematic Review |
P.142 |
Rasch Analysis of the Pediatric Quality of Life Inventory 4.0
Generic Core Scales Administered to Patients with Duchenne Muscular
Dystrophy |
P.345 |
Presentations will be archived under the events
and presentations section of the Sarepta Therapeutics website at
www.sarepta.com for one year following their presentation
at WMS25.
About Sarepta
TherapeuticsAt Sarepta, we are leading a revolution in
precision genetic medicine and every day is an opportunity to
change the lives of people living with rare disease. The Company
has built an impressive position in Duchenne muscular dystrophy
(DMD) and in gene therapies for limb-girdle muscular dystrophies
(LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth
(CMT), and other CNS-related disorders, with more than 40 programs
in various stages of development. The Company’s programs and
research focus span several therapeutic modalities, including RNA,
gene therapy and gene editing. For more information, please
visit www.sarepta.com or follow us on Twitter, LinkedIn,
Instagram and Facebook.
Internet Posting of
Information
We routinely post information that may be
important to investors in the 'For Investors' section of our
website at www.sarepta.com. We encourage investors and
potential investors to consult our website regularly for important
information about us.
Source: Sarepta Therapeutics, Inc.
Sarepta Therapeutics, Inc.
Investors: Ian Estepan, 617-274-4052, iestepan@sarepta.com
Media: Tracy Sorrentino, 617-301-8566,
tsorrentino@sarepta.com
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