Duchenne Research
Follow
Find
13.9K views | +2 today
 
Scooped by ParentProjectMD
onto Duchenne Research
Scoop.it!

Impact of Drisapersen Phase 3 Data on Prosensa Programs

Impact of Drisapersen Phase 3 Data on Prosensa Programs | Duchenne Research | Scoop.it
Prosensa has provided the following update regarding the impact of the drisapersen phase 3 data on its programs: 
While we are disappointed and surprised with…
more...
No comment yet.
Duchenne Research
Your new post is loading...
Your new post is loading...
Scooped by ParentProjectMD
Scoop.it!

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

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

PTC Therapeutics Reports Second Quarter Financial Results and Provides Corporate Update (NASDAQ:PTCT)

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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

ParentProjectMD's insight:

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

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

PTC Therapeutics to Host Conference Call to Discuss Second Quarter 2014 Financial Results (NASDAQ:PTCT)

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

PTC Therapeutics announces expanded access program for Translarna™ (ataluren)

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

International Study on the Burden of Duchenne Published in Neurology

International Study on the Burden of Duchenne Published in Neurology | Duchenne Research | Scoop.it
In the first international study of its kind, researchers have found that there are many different costs accompanying a rare condition such as Duchenne muscula…
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Akashi Therapeutics Receives Fast Track Designation for HT-100 from FDA for the Treatment of Duchenne Muscular Dystrophy

Akashi Therapeutics Receives Fast Track Designation for HT-100 from FDA for the Treatment of Duchenne Muscular Dystrophy | Duchenne Research | Scoop.it
From BioPortfolio: Akashi Therapeutics, Inc., announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the company�s most adv...
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Prosensa gets an FDA green light for its once-failed DMD drug

Prosensa gets an FDA green light for its once-failed DMD drug | Duchenne Research | Scoop.it
After a late-stage failure and a kiss-off from partner GlaxoSmithKline, Prosensa and its innovative treatment for Duchenne muscular dystrophy seemed to be on a downward slope.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Funding Opportunities-FY14 Duchenne Muscular Dystrophy Research Program, Congressionally Directed Medical Research Programs (CDMRP), US DoD

Funding Opportunities-FY14 Duchenne Muscular Dystrophy Research Program, Congressionally Directed Medical Research Programs (CDMRP), US DoD | Duchenne Research | Scoop.it
ParentProjectMD's insight:

Two DMDRP PAs have been posted on the CDMRP website.

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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.”

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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)

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Prosensa Holding N.V. to Webcast Conference Call Discussing 2nd Quarter 2014 Financial Results and Corporate Update on August 12, 2014 (NASDAQ:RNA)

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Exon-Skipping Trial Updates from Sarepta & Prosensa

Exon-Skipping Trial Updates from Sarepta & Prosensa | Duchenne Research | Scoop.it
In the wake of promising exon-skipping trial announcements earlier this summer, PPMD has been fielding many questions from the community about upcoming trial…
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Sarepta Therapeutics Inc - Investors - News Release

Sarepta Therapeutics Inc - Investors - News Release | Duchenne Research | Scoop.it
Sarepta Therapeutics Reports Long-Term Outcomes Through 144 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Summit presents new data from Phase 1b clinical trial of SMT C1100 for treatment of Duchenne

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Akashi Therapeutics Presents Positive Clinical Data on HT-100 in Patients with Duchenne

Akashi Therapeutics Presents Positive Clinical Data on HT-100 in Patients with Duchenne | Duchenne Research | Scoop.it
Preliminary Clinical Data from Ongoing Phase 1b/2a Clinical Program Presented at New Directions in Biology and Disease of Skeletal Muscle 6th Biennial Conference Cambridge, Mass.—July 3, 2014—Akashi Therapeutics, Inc., a...
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

PTC THERAPEUTICS APPOINTS RONALD C. RENAUD, JR. TO BOARD OF DIRECTORS

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Prosensa Announces A Regulatory Path Forward for Drisapersen as a Potential Treatment for Duchenne

Prosensa Announces A Regulatory Path Forward for Drisapersen as a Potential Treatment for Duchenne | Duchenne Research | Scoop.it
Prosensa Plans to Submit a New Drug Application to the FDA this Year; Dialogue with EMA continues with intent to seek approval

More good news from the FDA! Th…
more...
No comment yet.