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Prosensa Announces Drisapersen 48-Week Data and Recent Corporate Developments

Prosensa Announces Drisapersen 48-Week Data and Recent Corporate Developments | Duchenne Research | Scoop.it
Prosensa just released their 48-week data for drisapersen, as well as an update on recent corporate developments. Both press releases are included below.
PPMD…
ParentProjectMD's insight:

Prosensa just released their 48-week data for drisapersen, as well as an update on recent corporate developments. Both press releases are included.

PPMD has been talking with Prosensa and will be hosting a webinar with UPPMD later this month. We will be asking you for questions to pass along to Prosensa prior to the webinar. More to come soon.

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Milo Biotechnology's Follistatin Gene Therapy Increases Function in Becker Muscular Dystrophy Patients | DNA RNA and Cells | News Channels

Milo Biotechnology's Follistatin Gene Therapy Increases Function in Becker Muscular Dystrophy Patients | DNA RNA and Cells | News Channels | Duchenne Research | Scoop.it
Milo Biotechnology's Follistatin Gene Therapy Increases Function in Becker Muscular Dystrophy Patients
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Immune Cells May Help Treat Muscular Dystrophy, UCSF-Led Team Finds | ucsf.edu

Immune Cells May Help Treat Muscular Dystrophy, UCSF-Led Team Finds | ucsf.edu | Duchenne Research | Scoop.it

A scientific team led by UC San Francisco researchers found that regulatory T cells (Tregs), a specialized subset of immune cells, suppress inflammation and muscle injury in a mouse model of Duchenne muscular dystrophy (DMD).

 

The scientists said that Tregs have potential as therapeutic agents for DMD, an inherited disease that strikes children – almost always boys – and leads to progressive muscle degeneration and early death.

 

They published their findings online October 15, 2014, in Science Translational Medicine.

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New research suggests strategy for reducing muscle inflammation in Duchenne.

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News from World Muscle Society: Days 2 & 3

News from World Muscle Society: Days 2 & 3 | Duchenne Research | Scoop.it
PTC Therapeutics Symposium – “Improving Outcomes for the Future: Making sense of the nonsense mutations in Duchenne muscular dystrophy”

PTC Therapeutics gave…
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Prosensa begins NDA submission to the FDA for exon-skipping drug drisapersen to treat Duchenne muscular dystrophy (NASDAQ:RNA)

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Capricor Announces Plans to Pursue Clinical Program for the Treatment of Duchenne Muscular Dystrophy With Cardiosphere-Derived Cells (CDCs)

Capricor Announces Plans to Pursue Clinical Program for the Treatment of Duchenne Muscular Dystrophy With Cardiosphere-Derived Cells (CDCs) | Duchenne Research | Scoop.it
LOS ANGELES, Oct. 6, 2014 (GLOBE NEWSWIRE) --
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Santhera to Present Results from Phase III Study with Catena®/ Raxone® in Duchenne Muscular Dystrophy at the International Congress of the World Muscle Society

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Prosensa announces commencement of re-dosing of drisapersen in North America in patients with Duchenne muscular dystrophy

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Gene therapy protects mice from lethal heart condition, researchers find

Date:

August 20, 2014

Source:

University of Missouri-Columbia

Summary:

A new gene therapy has been shown to protect mice from a life-threatening heart condition caused by muscular dystrophy. About one in 3,500 children, mostly boys, are born with Duchenne muscular dystrophy (DMD). They experience a progressive wasting away of muscles, starting in the legs and pelvis. Children with DMD have difficulty walking, and most need wheelchairs by age 12.

 

A new gene therapy developed by researchers at the University of Missouri School of Medicine has shown to protect mice from a life-threatening heart condition caused by muscular dystrophy.

"This is a new therapeutic avenue," said Yi Lai, PhD, the leading author of the study and assistant research professor in the MU School of Medicine's Department of Molecular Microbiology and Immunology. "This is just a first step, but we hope this could lead to a treatment for people with this devastating heart condition, which is a leading cause of death for people with Duchenne muscular dystrophy."

About one in 3,500 children, mostly boys, are born with Duchenne muscular dystrophy (DMD). They experience a progressive wasting away of muscles, starting in the legs and pelvis. Children with DMD have difficulty walking, and most need wheelchairs by age 12.

As DMD depletes the skeletal muscles, it also causes the heart to decay. A weakened heart kills up to 40 percent of people with DMD, usually by their 20s or early 30s. DMD originates with mutations in a single gene. For more than two decades, researchers have explored using gene therapy, an experimental treatment, to replace the flawed gene with a healthy copy.

The recent MU study, however, did not try to replace the faulty gene. The researchers targeted a different gene -- one involved with the heart's built-in system for responding to heart attacks and other emergencies.

This targeted gene expresses a protein called nNOS. During short-term stresses, nNOS activates briefly to help regulate the heart. The MU researchers altered the gene to enable more efficient transfer of the nNOS gene to mouse hearts.

Seven months after the gene therapy, the mice who received the treatment showed significantly improved overall heart health. On most disease indicators, the researchers found that the treatment protected their hearts from the damage of DMD.

"The study showed for the first time that a modified nNOS gene could be delivered through gene therapy to protect the hearts of mice from Duchenne muscular dystrophy," said Dongsheng Duan, PhD, co-author of the study and Margaret Proctor Mulligan Professor in Medical Research at the MU School of Medicine.

"Since nNOS protects against multiple heart diseases, this method could one day be extended to the treatment of other heart diseases, such as heart failure or a heart attack," Duan said.

The technique is in an early stage of development and will require more research before potential applications in humans are explored.

 

Story Source:

The above story is based on materials provided by University of Missouri-Columbia.Note: Materials may be edited for content and length.

 

Journal Reference:

1.     Y. Lai, J. Zhao, Y. Yue, N. B. Wasala, D. Duan. Partial restoration of cardiac function with  PDZ nNOS in aged mdx model of Duchenne cardiomyopathy.Human Molecular Genetics, 2014; 23 (12): 3189 DOI: 10.1093/hmg/ddu029

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PTC THERAPEUTICS COMPLETES ENROLLMENT OF LANDMARK TRIAL IN DUCHENNE MUSCULAR DYSTROPHY (NASDAQ:PTCT)

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Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy

Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy | Duchenne Research | Scoop.it
New technology designed to speed dystrophin measurement while ensuring consistencyAutomation of dystrophin measurement will aid development of Sarepta’s Duchenne muscular dystrophy candidates
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PTC Therapeutics Reports Second Quarter Financial Results and Provides Corporate Update (NASDAQ:PTCT)

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PTC Therapeutics Receives Conditional Approval in the European Union for Translarna™ For the Treatment of Nonsense Mutation Duchenne Muscular Dystrophy (NASDAQ:PTCT)

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The European Commission has granted accelerated approval for Translarna within the European Union, as predicted based on an earlier recommendation by the CHMP.

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Sarepta Therapeutics to Announce Second Quarter 2014 Financial Results and Corporate Update on August 7, 2014

Sarepta Therapeutics to Announce Second Quarter 2014 Financial Results and Corporate Update on August 7, 2014 | Duchenne Research | Scoop.it
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UB-Developed Spider-Venom Drug for Muscular Dystrophy Progresses - UB School of Medicine and Biomedical Sciences

UB-Developed Spider-Venom Drug for Muscular Dystrophy Progresses - UB School of Medicine and Biomedical Sciences | Duchenne Research | Scoop.it
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News from World Muscle Society: Day 4

News from World Muscle Society: Day 4 | Duchenne Research | Scoop.it
Late Breaking Talks:

PPMD-funded gene therapy study improves walking ability in muscular dystrophy
Dr. Jerry Mendell of Nationwide Children’s Research Instit…
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News from World Muscle Society Meeting (10/8/14)

News from World Muscle Society Meeting (10/8/14) | Duchenne Research | Scoop.it
Sarepta held a symposium on the role of dystrophin production in disease-modifying treatments for Duchenne. Some highlights:
 
We heard from Dr. Francesco Mun…
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Summit Reports Positive Data on it Utrophin Modulation Programme for the Treatment of DMD at 2014 WMS Congress

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Prosensa extends the re-dosing of drisapersen in Europe in patients with Duchenne muscular dystrophy (NASDAQ:RNA)

Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that it has extended its comprehensive program of re-dosing of drisapersen in  patients with Duchenne muscular dystrophy (DMD) into Europe, with the PRO051-02/DMD114673 sites re-opening, beginning withBelgium. This follows quickly on the re-dosing program that began in the United States (US) last week.

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Prosensa publishes a sensitive, reproducible and objective methodology for dystrophin analysis in patients with Duchenne muscular dystrophy (NASDAQ:RNA)

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PTC Therapeutics Expands Global Presence in Support of Translarna™ Launch in Duchenne Muscular Dystrophy (NASDAQ:PTCT)

PTC Therapeutics Expands Global Presence in Support of Translarna™ Launch in Duchenne Muscular Dystrophy (NASDAQ:PTCT) | Duchenne Research | Scoop.it
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Akashi Therapeutics Acquires Global Rights to Novel DMD Treatment from Tonus Therapeutics - Akashi RX

Akashi Therapeutics Acquires Global Rights to Novel DMD Treatment from Tonus Therapeutics - Akashi RX | Duchenne Research | Scoop.it

GsMTx-4 Addresses Calcium Level Imbalance – a Critical Area of Need in DMD

Akashi Therapeutics Inc., a clinical stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy (DMD),announced today that it has acquired global rights to GsMTx-4, a peptide developed by Tonus Therapeutics to address calcium level imbalance in muscle, a critical issue in DMD contributing to loss of function and other associated pathologies. Originally discovered in tarantula venom by researchers at the State University of New York at Buffalo, GsMTx-4 has been shown to positively affect cellular calcium homeostasis in preclinical DMD model studies generated by Tonus.

Under the terms of the agreement, Akashi Therapeutics will acquire global rights to the compound, including intellectual property and commercialization rights,and will be responsible for all ongoing development costs. Tonus will be eligible to receive potential milestones and royalties on future sales of any resulting DMD products. No further terms were disclosed.

“Loss of calcium homeostasis, in particular increased calcium influx through stretch-activated channels,in muscle cells of DMD boys is a key initiating process of DMD pathology leading to muscle degeneration and muscle function loss,” said Professor Urs Ruegg, Ph.D., Department of Pharmacology at the University of Geneva. “We know that limiting calcium influx has the potential to slow disease progression. As GsMTx-4 is a blocker of stretch-activated channels, it has the potential to help restore this homeostasis through modulation of these channels.”

“Calcium level imbalance and associated muscle function loss is a critical problem facing children with DMD and an area that is not being fully addressed by other DMD therapies in development,” said Marc B. Blaustein, CEO of Akashi Therapeutics. “Our mission at Akashi Therapeutics is to develop a portfolio of treatments for Duchenne muscular dystrophy. We are pleased to add GsMTx-4 to our growing pipeline, which includes HT-100, our most advanced drug candidate, currently being evaluated in patients with DMD in phase 1a/2b clinical studies, and DT-200, a clinical-stage selective androgen receptor modulator.”

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Prosensa announces Lancet Neurology publication of an exploratory phase II study (DEMAND II) demonstrating efficacy and safety of drisapersen in patients with Duchenne muscular dystrophy (NASDAQ:RNA)

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Prosensa Announces 2nd Quarter 2014 Financial Results and Recent Corporate Developments (NASDAQ:RNA)

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Sarepta Therapeutics Announces Second Quarter 2014 Financial Results and Recent Corporate Developments

Sarepta Therapeutics Announces Second Quarter 2014 Financial Results and Recent Corporate Developments | Duchenne Research | Scoop.it
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Prosensa Holding N.V. to Webcast Conference Call Discussing 2nd Quarter 2014 Financial Results and Corporate Update on August 12, 2014 (NASDAQ:RNA)

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