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Prosensa to Host Conference Call to Discuss Recent Drisapersen Data Presentations

Prosensa will hold a conference call on Tuesday, October 8, 2013 at 11:00 a.m. ET to discuss recently-presented data on drisapersen, an investigational antisense oligonucleotide, for the treatment of Duchenne Muscular Dystrophy patients with an amenable mutation, which is exclusively licensed to GlaxoSmithKline (GSK).


Prosensa Chief Executive Officer Hans Schikan will be joined by Giles Campion, Prosensa's Chief Medical Officer & Senior Vice-President of Research and Development and Judith van Deutekom, Vice President of Drug Discovery to discuss the recently presented results from studies of drisapersen. These include results from DEMAND III (Phase III; DMD114044); DEMAND II (Phase II; DMD114117); DEMAND V (Phase II; DMD114876) and 177 week data from the Phase I/II extension study (DMD114673).

 

In order to participate in the conference call, please dial 1-877 280 2342 (US domestic) and refer to conference ID 2579927. International dial-in numbers and an audio webcast can be accessed under "Events & Presentations" through the Investors & Media section of the Prosensa corporate website www.prosensa.com.

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PTC Therapeutics Announces CEO, Dr. Stuart Peltz Recipient of The New Jersey Technology Council's 2014 Legend of Technology Award (NASDAQ:PTCT)

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Capricor Therapeutics Announces Positive Pre-Clinical Data for Cardiosphere-Derived Cells (CDCs) on Duchenne Muscular Dystrophy Cardiomyopathy

Capricor Therapeutics Announces Positive Pre-Clinical Data for Cardiosphere-Derived Cells (CDCs) on Duchenne Muscular Dystrophy Cardiomyopathy | Duchenne Research | Scoop.it
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Prosensa Announces 3rd Quarter 2014 Financial Results and Recent Corporate Developments (NASDAQ:RNA)

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Pfizer Myostatin Blocker Trial Set to Launch

Pfizer Myostatin Blocker Trial Set to Launch | Duchenne Research | Scoop.it
At the Action Duchenne meeting in London on Saturday, Pfizer Inc. announced that it has completed a study in healthy volunteers with its antibody-based thera…
ParentProjectMD's insight:

Sharon Hesterlee and Annie Kennedy from the PPMD team recently attended the Action Duchenne Conference in London. At the meeting, Pfizer announced that it has completed a study in healthy volunteers with its antibody-based therapeutic to block the muscle protein myostatin, and is preparing to start recruitment for a phase II study in Duchenne this month. Read Sharon and Annie’s report to learn more.

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Idera Pharmaceuticals' (IDRA) CEO Sudhir Agrawal on Q3 2014 Results - Earnings Call Transcript

Idera Pharmaceuticals' (IDRA) CEO Sudhir Agrawal on Q3 2014 Results - Earnings Call Transcript | Duchenne Research | Scoop.it
Idera Pharmaceuticals, Inc. (NASDAQ:IDRA) Q3 2014 Earnings Conference Call November 7, 2014 08:00 AM ET Executives Jim Baker - Executive Director of Corporate Affairs Sudhir Agrawal - CEO Lou Arcudi - CFO Lou Brenner - CMO Ka
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Capricor Therapeutics Reports Third Quarter 2014 Financial & Business Highlights

Capricor Therapeutics Reports Third Quarter 2014 Financial & Business Highlights | Duchenne Research | Scoop.it
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PTC Therapeutics' (PTCT) CEO Stuart Peltz on Q3 2014 Results - Earnings Call Transcript

PTC Therapeutics' (PTCT) CEO Stuart Peltz on Q3 2014 Results - Earnings Call Transcript | Duchenne Research | Scoop.it
PTC Therapeutics, Inc. (NASDAQ:PTCT) Q3 2014 Earnings Conference Call November 6, 2014 09:00 ET Executives Emily Hill - Director, Investor Relations Stuart Peltz - Chief Executive Officer Shane Kovacs - Chief Financial Office
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Cambridge biotech Idera jumps into Duchenne muscular dystrophy field - Boston Business Journal

Cambridge biotech Idera jumps into Duchenne muscular dystrophy field - Boston Business Journal | Duchenne Research | Scoop.it
The biotech has formed a partnership with Parent Project Muscular Dystrophy to conduct early stage research into a drug which would slow the deterioration of muscles caused by Duchenne.
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GEN | Insight & Intelligence™: Community Organizing Meets the Clinic

GEN | Insight & Intelligence™: Community Organizing Meets the Clinic | Duchenne Research | Scoop.it
Registries connect patients with advocates, clinicians, and drug developers.
ParentProjectMD's insight:

As DuchenneConnect turns 7 this year, we are thrilled to see the way this resource has changed the landscape of patient registries. We often hear from families and companies that DuchenneConnect has helped patients and clinical trials find each other. Not only is this site benefiting the Duchenne community, though, it has created a model that other health registries look to. 

If you have not registered on DuchenneConnect or if you have not updated your information in a while, visit http://DuchenneConnect.org today!

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

Sarepta Therapeutics Announces Third Quarter 2014 Financial Results and Recent Corporate Developments | Duchenne Research | Scoop.it

- Eteplirsen confirmatory study in ambulatory patients and study in advanced/non-ambulatory patients to begin dosing this month -

- New Drug Application submission for eteplirsen planned for mid-year 2015 based on recent FDA guidance -

- Revised 2014 non-GAAP operating loss guidance lowered to $110-$120 million with cash and other investments of $240 million at quarter end -

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Milo Biotechnology makes headway on reversing disease

Milo Biotechnology makes headway on reversing disease | Duchenne Research | Scoop.it
Al Hawkins thinks he has found a way to reverse muscular dystrophy. It's too early to say if he's right, but new evidence suggests that he could be.
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Translarna™ Phase 2b Data Published in Muscle & Nerve

Data Supports Benefit in Nonsense Mutation Duchenne Muscular Dystrophy Patients
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Milo Biotechnology's Follistatin Gene Therapy Increases Function in Becker Muscular Dystrophy Patients

Milo Biotechnology's Follistatin Gene Therapy Increases Function in Becker Muscular Dystrophy Patients | Duchenne Research | Scoop.it
Milo Biotechnology's Follistatin Gene Therapy Increases Function in Becker Muscular Dystrophy Patients
ParentProjectMD's insight:

PPMD-funded gene therapy study improves walking ability in Becker muscular dystrophy.


Dr. Mendell is currently planning a similar trial in Duchenne muscular dystrophy, but will plan to inject the follistatin gene into gluteal muscles, thigh muscles and the tiabialis anterior muscles because all of these muscle groups are strongly affected in Duchenne. He speculates that even muscle groups that are not injected directly may show some benefit since follistatin is secreted and can circulate through the body. Animal studies showed evidence of these “remote effects” away from the site of injection.

As always, PPMD will keep you up to date on the next steps in this exciting research.
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Sarepta Therapeutics Announces First Patient Dosed in Confirmatory Study of Eteplirsen in Ambulant Patients with Duchenne Muscular Dystrophy

Sarepta Therapeutics Announces First Patient Dosed in Confirmatory Study of Eteplirsen in Ambulant Patients with Duchenne Muscular Dystrophy | Duchenne Research | Scoop.it
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PTC Therapeutics Appoints Dr. Tuyen Ong SVP, Head of Clinical Development and Translational Research (NASDAQ:PTCT)

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PTC Therapeutics Recognized for Outstanding Innovation at the 2014 Rare & Orphan Advocacy and Research (ROAR) Awards (NASDAQ:PTCT)

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Sarepta Therapeutics Announces First Patient Dosed in Study of Eteplirsen in Non-Ambulant Patients with Duchenne Muscular Dystrophy

Sarepta Therapeutics Announces First Patient Dosed in Study of Eteplirsen in Non-Ambulant Patients with Duchenne Muscular Dystrophy | Duchenne Research | Scoop.it

Sarepta Therapeutics, Inc., a developer of RNA-based therapeutics, today announced that it has initiated dosing in a clinical study of eteplirsen, the Company's lead exon-skipping therapeutic candidate for the treatment of Duchenne muscular dystrophy (DMD), in patients who are non-ambulant or who have advanced DMD and don't meet a minimum 6-minute walk test score at baseline.

 

The open-label study, 4658-204 (Study 204), will include approximately 20 patients treated with eteplirsen who have genotypes amenable to exon 51 skipping and who meet other study inclusion criteria. The study will be conducted at several sites in the United Statesand is designed to evaluate the safety of eteplirsen in DMD patients over 96 weeks of dosing. Patients enrolled in the study will receive once weekly intravenous infusions of 30mg/kg of eteplirsen, and data will be collected across a number of safety parameters and secondary efficacy endpoints.

 

“The initiation of this eteplirsen study represents an important milestone for patients, their families, and the DMD community,” said Edward Kaye, M.D., Sarepta’s Chief Medical Officer. “Expanding the DMD population to include patients who are older and non-ambulant demonstrates our strong commitment to develop eteplirsen for patients at all stages of DMD and will provide additional data to support our planned NDA filing.”

 

Fawn Leigh, M.D., of Mass General Hospital and a principal investigator in the study added, "Eteplirsen is a potential breakthrough treatment for patients with DMD. I am pleased to be able to offer this promising disease-modifying treatment to my patients and to potentially alter the course of this devastating disease.”

 

 

About Eteplirsen

Eteplirsen is Sarepta's lead drug candidate and is designed to address the underlying cause of DMD by enabling the production of a functional dystrophin protein. Data from clinical studies of eteplirsen in DMD patients have demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression.

Eteplirsen uses Sarepta's novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene enabling the repair of specific genetic mutations that affect approximately 13 percent of the total DMD population. By skipping exon 51, eteplirsen may restore the gene's ability to make a shorter, but still functional, form of dystrophin from messenger RNA, or mRNA. Promoting the synthesis of a truncated dystrophin protein is intended to stabilize or significantly slow the disease process and prolong and improve the quality of life for patients with DMD. Sarepta is also developing other PMO-based exon-skipping drug candidates intended to treat additional patients with DMD.

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Sarepta Therapeutics' (SRPT) CEO Chris Garabedian on 23rd Annual Credit Suisse Healthcare Conference Call Transcript

Sarepta Therapeutics' (SRPT) CEO Chris Garabedian on 23rd Annual Credit Suisse Healthcare Conference Call Transcript | Duchenne Research | Scoop.it
Sarepta Therapeutics Inc. (NASDAQ:SRPT) 23rd Annual Credit Suisse Healthcare Conference Call November 11, 2014 6:00 PM ET Executives Christopher Garabedian – Chief Executive Officer and President Analysts Jeremiah B. Shepard
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Sarepta’s Safety Study of Eteplirsen to Treat Advanced Stage Duchenne has Initiated Recruitment

Sarepta’s Safety Study of Eteplirsen to Treat Advanced Stage Duchenne has Initiated Recruitment | Duchenne Research | Scoop.it

One site in the U.S. is now actively recruiting for this trial, and Sarepta is working towards the goal of having 9 sites participate in this trial. They plan to continue initiating study sites on a rolling basis between now and early 2015. 

 

The primary objective of this study is to explore safety and tolerability of eteplirsen in patients with advanced stage Duchenne who are amenable to exon 51 skipping. The exploratory objectives are to evaluate the effect of eteplirsen on pulmonary function tests (PFTs) and other functional clinical measures. The study sponsor is Sarepta Therapeutics.

 

Recruitment Criteria:

- Males with a diagnosis Duchenne and a genetic mutation amenable to exon 51 skipping

- Ages 7-21 years old

- Stable corticosteroid therapy for at least 6 months, or no corticosteroid therapy for 6 months

- Non-ambulatory (not able to walk ≥300 meters on the 6-Minute Walk Test)

- Stable pulmonary and cardiac function (predicted FVC equal to or greater than 40% and LVEF equal to or greater than 40%)

- Please see the ClinicalTrials.gov summary for additional inclusion and exclusion criteria

 

The estimated number of participants is 20.  All participants will receive once weekly intravenous (IV) infusions of eteplirsen 30 mg/kg for 96 weeks. 

 

Safety will be regularly assessed throughout the study via the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations. Exploratory efficacy assessments, including PFTs, upper extremity testing, and other measurements of functional status, will occur at functional assessment visits every 12 weeks over the first year of treatment and approximately every 24 weeks over the second year of treatment.

 

 For additional information, including a list of all trial sites, please read the trial summary posted on ClinicalTrials.gov.

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Capricor Therapeutics to Hold Duchenne Muscular Dystrophy Data Presentation Call on November 18, 2014 at 9:30 a.m. ET

Capricor Therapeutics to Hold Duchenne Muscular Dystrophy Data Presentation Call on November 18, 2014 at 9:30 a.m. ET | Duchenne Research | Scoop.it
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Idera Pharmaceuticals and Parent Project Muscular Dystrophy Announce Collaboration to Advance New Treatment Approach for Duchenne Muscular Dystrophy

Idera Pharmaceuticals and Parent Project Muscular Dystrophy Announce Collaboration to Advance New Treatment Approach for Duchenne Muscular Dystrophy | Duchenne Research | Scoop.it
Toll-like receptor antagonism designed to treat muscle inflammation central to disease progressionNew approach offers potential to treat broad population of patients regardless of their genetic mutation
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PTC Therapeutics Reports Third Quarter Financial Results and Provides Corporate Update

PTC Therapeutics Reports Third Quarter Financial Results and Provides Corporate Update | Duchenne Research | Scoop.it
- Confirmatory Phase 3 ACT DMD clinical trial fully enrolled -
- Rolling NDA submission for Translarna in nmDMD planned before year end 2014 -
- European commercial launch activities well underway -
- Initiation of Phase 1b/2a trial for SMA program planned -
- IND application for PTC596 to be submitted before year end 2014 -
- Successful financing raised estimated net proceeds of $117.5 million -
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PTC Therapeutics Expands Access For Translarna™ (Ataluren)

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Translarna™ (ataluren) has been approved to be included in the list of drugs reimbursable by the Italian National Health System under Law 648/96. The decision is expected to be formalized through its publication in the official Italian journal, Gazzetta Ufficiale in the coming weeks. A list of drugs reimbursable under this law is published on the Italian Medicines Agency's (AIFA) website.

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PPMD Submits Letter to FDA Regarding Latest Sarepta Update

PPMD Submits Letter to FDA Regarding Latest Sarepta Update | Duchenne Research | Scoop.it
Today’s press release and investor call from Sarepta have sent a ripple of angst and sadness through our community. Moments ago, PPMD sent a letter to the FDA…
ParentProjectMD's insight:

Today’s press release and investor call from Sarepta (http://bit.ly/1xwCbi1) have sent a ripple of angst and sadness through our community.

Moments ago, PPMD sent a letter to the FDA that reflects our organization’s belief that safety and rigor have been demonstrated throughout this process. Further, we believe that any strengthening of data packages that causes delays (as is now being requested by the FDA, as articulated in today’s press release and call) should ensure a streamlined, more expeditious approval process for follow-on exons.

As a community we believe exon-skipping drugs like eteplirsen hold potentially life-altering benefits, and must be moved forward as rapidly as possible. In addition, we believe eteplirsen to be a critical foundational therapy, restoring dystrophin and significantly slowing disease progression. We look forward to the mid-2015 submission and approval of eteplirsen and the consideration of approval of PMO (phosphodiamidate morpholino oligomer) as a class of drugs that will have the ability to slow/halt progression in a great many of the individuals living with Duchenne.

PPMD has also reached out to leadership at Sarepta to support the pathway forward for these trials and to work to ensure the approval and availability of an effective therapy to our Duchenne community at the earliest moment possible. As always we will report back to you with updates on next steps and anything we as a community can do to help this process.

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Sarepta Therapeutics Announces Regulatory Update on Eteplirsen

Sarepta Therapeutics Announces Regulatory Update on Eteplirsen | Duchenne Research | Scoop.it

Updated and additional guidance received from FDA on specific data requirements for NDA;

FDA states further discussion needed to determine what constitutes a “complete” NDA submission;

NDA submission planned for mid-year 2015;

Company to hold teleconference today at 8:00 a.m. EDT

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