Duchenne Muscular Dystrophy Research
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Impact of Drisapersen Phase 3 Data on Prosensa Programs

Impact of Drisapersen Phase 3 Data on Prosensa Programs | Duchenne Muscular Dystrophy 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…
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PPMD Awards $600,000 Grant to NJIT & Talem for Next Phase of Upper Extremity Exoskeleton

PPMD Awards $600,000 Grant to NJIT & Talem for Next Phase of Upper Extremity Exoskeleton | Duchenne Muscular Dystrophy Research | Scoop.it
Robotics.
 
Ensuring that the worlds of teens and adults with Duchenne 'grow bigger', as they grow older by expanding their reach – both literally and figura…
ParentProjectMD's insight:
PPMD has awarded a $600,000 grant to the New Jersey Institute of Technology (NJIT) and Talem Technologies as part of our ongoing exploration of robotic technology to assist people living with Duchenne.
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Akashi Therapeutics Provides Update on Three Duchenne Compounds

Akashi Therapeutics Provides Update on Three Duchenne Compounds | Duchenne Muscular Dystrophy Research | Scoop.it
This weekend, Akashi Therapeutics announced they are working toward having three novel, complementary compounds in the clinic in 2017 with potential to trea…
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Sarepta Therapeutics Enters into Research Agreement and Option Agreement with Nationwide Children’s Hospital for Microdystrophin Gene Therapy Program

Sarepta Therapeutics Enters into Research Agreement and Option Agreement with Nationwide Children’s Hospital for Microdystrophin Gene Therapy Program | Duchenne Muscular Dystrophy Research | Scoop.it
On the heels of PPMD’s announcement last week of a $2.2 million grant to Nationwide Children’s Hospital’s gene therapy study being led by Dr. Jerry Mendell and…
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Invitae acquires patient-centered data company AltaVoice, creating new offerings to advance research and access to care for patients with inherited and rare diseases

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“Genetic testing is essential in diagnosing rare diseases, but the combination of genetic information with patient-reported data has potential well beyond diagnosis to help advance the understanding and treatment of these diseases,” said Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy. “We are heartened to see AltaVoice and Invitae joining forces to help forge new ways to connect patients to care and to contribute to vital research.”
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Santhera’s Raxone® designated Promising Innovative Medicine (PIM) and suitable candidate for further evaluation under the UK Early Access to Medicines Scheme (EAMS) for treatment in Duchenne muscul...

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[Webinar] A Closer Look at the Potential of CRISPR/Cas9 in Duchenne - December 2016

On December 15, 2016, PPMD hosted a webinar with Dr. Eric Olson from the Department of Molecular Biology at UT Southwestern Medical Center to review th
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Duchenne Carrier Study at Nationwide Children’s Hospital

Duchenne Carrier Study at Nationwide Children’s Hospital | Duchenne Muscular Dystrophy Research | Scoop.it
Frequently, I get calls from mothers/sisters/aunts of people living with Duchenne about issues related to being a carrier of Duchenne:

Should they have carr…
ParentProjectMD's insight:
There is widespread consensus that female carriers of Duchenne are at risk for symptoms of muscular dystrophy. But what are those risks? PPMD is excited to support this important study at Nationwide Children’s Hospital, which will evaluate the effects of a lower level of dystrophin in female carriers in three meaningful areas—cardiac, musculoskeletal, and psychosocial. We hope the results of this study will help us better understand the implications of being a Duchenne carrier, and possibly apply some of the answers to our understanding of Duchenne in males.
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Capricor Therapeutics Reports Third Quarter 2016 Financial Results and Provides Corporate Update

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Pfizer Shares Update on Phase II Clinical Trial of Domagrozumab (PF-06252616) in Duchenne

Pfizer Shares Update on Phase II Clinical Trial of Domagrozumab (PF-06252616) in Duchenne | Duchenne Muscular Dystrophy Research | Scoop.it
Pfizer has provided the following update regarding the on-going Phase II clinical trial of domagrozumab (PF-06252616), a myostatin inhibitor, in people with Du…
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Solid Biosciences Granted U.S. and E.U. Orphan Drug Designations for Lead Gene Therapy Candidate for Duchenne Muscular Dystrophy

Solid Biosciences Granted U.S. and E.U. Orphan Drug Designations for Lead Gene Therapy Candidate for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
Solid Biosciences and its subsidiary, Solid GT, announced today that the FDA and the European Commission have granted Orphan Drug designations for the compa…
ParentProjectMD's insight:
Solid Biosciences and its subsidiary, Solid GT, announced today that the FDA and the European Commission have granted Orphan Drug designations for the company’s gene therapy candidate, SGT-001, for the treatment of people with Duchenne. Solid plans to initiate clinical studies for SGT-001 in 2017.
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Duchenne Research Update: October 2016

Duchenne Research Update: October 2016 | Duchenne Muscular Dystrophy Research | Scoop.it
When I first joined the Duchenne community in 2009 at Children’s National Medical Center, I remember thinking that I was walking into a critical juncture in…
ParentProjectMD's insight:
So much has happened in recent weeks in the Duchenne space that it can be hard to keep up. While approvals and regulatory actions have dominated the press, there is also a lot of pre-clinical and early stage clinical research happening behind the scenes with great potential. PPMD’s Abby Bronson, SVP, Research Strategy provides a roundup of the latest Duchenne research news.
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PTC Therapeutics Announces New Analyses that Support the Potential Benefit of Ataluren in Preserving Lung Function in Non-Ambulatory Nonsense Mutation Duchenne Muscular Dystrophy Patients

PTC Therapeutics Announces New Analyses that Support the Potential Benefit of Ataluren in Preserving Lung Function in Non-Ambulatory Nonsense Mutation Duchenne Muscular Dystrophy Patients | Duchenne Muscular Dystrophy Research | Scoop.it
PTC Therapeutics, Inc. today announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory people with nonsens…
ParentProjectMD's insight:
PTC Therapeutics, Inc. today announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory people with nonsense mutation Duchenne. PPMD continues to urge the FDA to provide PTC with a full review and advisory committee meeting.
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Deb Robins's curator insight, November 14, 2016 12:47 PM
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New Research Validates Translarna's™ (ataluren) Mechanism of Action to Promote Readthrough of Nonsense Mutations and Produce Full-length Functional Protein

New Research Validates Translarna's™ (ataluren) Mechanism of Action to Promote Readthrough of Nonsense Mutations and Produce Full-length Functional Protein | Duchenne Muscular Dystrophy Research | Scoop.it
PTC Therapeutics announced that the Proceedings of the National Academy of Sciences (PNAS) has published new results further validating Translarna's™ (atalur…
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Catabasis Pharmaceuticals Phase 1 Data on Edasalonexent (CAT-1004), a Potential Disease-Modifying Therapy Being Developed for Duchenne Muscular Dystrophy, Published in the Journal of Clinical Pharm...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 19, 2017-- Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the publication of Phase 1 data on edasalonexent in adult subjects. Edasalonexent is a potential disease-modifying therapy being developed for Duchenne muscular dystrophy (DMD). The Phase 1 trials demonstrated that edasalonexent (CAT-1004), an oral inhibitor of NF-kB, was safe, well tolerated, and inhibited activated NF-kB in adult subjects and the data are presented in an article titled “A Novel NF-kB Inhibitor, Edasalonexent (CAT-1004), in Development as a Disease-Modifying Treatment for Patients with Duchenne Muscular Dystrophy: Phase 1 Safety, Pharmacokinetics, and Pharmacodynamics in Adult Subjects” in the Journal of Clinical Pharmacology (J Clin Pharmacol. 2017 Jan 11. doi: 10.1002/jcph.842.)

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Sarepta Therapeutics Enters into License Agreement with Nationwide Children’s Hospital for Galgt2 Gene Therapy Program

Sarepta Therapeutics Enters into License Agreement with Nationwide Children’s Hospital for Galgt2 Gene Therapy Program | Duchenne Muscular Dystrophy Research | Scoop.it
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PPMD Awards Nationwide Children’s Hospital $2.2 Million Grant to Explore Gene Therapy in Duchenne

PPMD Awards Nationwide Children’s Hospital $2.2 Million Grant to Explore Gene Therapy in Duchenne | Duchenne Muscular Dystrophy Research | Scoop.it
Today, PPMD announced a $2.2 million dollar grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac (co-PI), and Nationwide Children’s Hospital.  Our purpose in…
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Positive Preclinical Research on the Edasalonexent (CAT-1004) Program, a Potential Disease-Modifying Therapy for Duchenne Muscular Dystrophy, Published in JCI Insight

Catabasis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, today announced the publication of preclinical data on the edasalonexent program, a potential disease-modifying therapy for Duchenne muscular dystrophy (DMD). The preclinical data demonstrate that edasalonexent (CAT-1004) and an analog, CAT-1041, oral inhibitors of NF-kB, are effective in ameliorating the dystrophic process in two animal models of DMD in an article titled “Disease Modifying Effects of Orally Bioavailable NF-kB Inhibitors in Dystrophin-Deficient Muscle” in JCI Insight (JCI Insight 2016 Dec 22;1(21):e90341).

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Sarepta Therapeutics Announces EMA Validation of Eteplirsen Authorization Application for Treatment of Duchenne Muscular Dystrophy Amenable to Exon Skipping 51

Sarepta Therapeutics Announces EMA Validation of Eteplirsen Authorization Application for Treatment of Duchenne Muscular Dystrophy Amenable to Exon Skipping 51 | Duchenne Muscular Dystrophy Research | Scoop.it
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PTC Therapeutics Receives France's 2016 Prix Galien for Translarna™

SOUTH PLAINFIELD, N.J., Dec. 8, 2016 /PRNewswire/-- PTC Therapeutics Inc. (NASDAQ: PTCT), today announced that the company received France's 2016 Prix Galien for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in the Medicines "Rare Disease" category. The award recognizes the scientific innovation represented by Translarna's ability to target the underlying cause of nmDMD and the impact the drug is having on patients. The award was presented at the annual gala and ceremony in Paris, France.

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CHMP Recommends Renewal of Translarna's™ Marketing Authorization Based on Continued Positive Benefit-Risk Assessment

CHMP Recommends Renewal of Translarna's™ Marketing Authorization Based on Continued Positive Benefit-Risk Assessment | Duchenne Muscular Dystrophy Research | Scoop.it
PTC Therapeutics, Inc. today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended t…
ParentProjectMD's insight:
PTC Therapeutics today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne. As a condition of the renewal, PTC is required to conduct an additional long-term post-authorization trial.

While PTC's regulatory engagement with the EMA is separate from the engagement with U.S. regulators at the FDA, today’s decision will enable the continuation of global access to Translarna and powerful data collection to further support PTC's pursuit of an access pathway to Translarna for U.S. patients. PPMD is continuing to work closely with PTC and is pleased that European authorities are recognizing the importance of benefit-risk assessments within product reviews.
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Actelion obtains an option to in-license vamorolone from ReveraGen

Actelion obtains an option to in-license vamorolone from ReveraGen | Duchenne Muscular Dystrophy Research | Scoop.it
Actelion obtains an option to in-license vamorolone from ReveraGen
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The Latest Update from Translarna

The Latest Update from Translarna | Duchenne Muscular Dystrophy Research | Scoop.it
The journey has been long. From PTC Therapeutics' first presentation about targeting a ‘stop sign’ within the dystrophin mutation to PTC 124 to ataluren and…
ParentProjectMD's insight:
PPMD continues to believe that our Duchenne community deserves a full and fair review of the Translarna data by scientific experts at an Advisory Committee Meeting. Your personal experiences living with Duchenne and participating in clinical research are critical if we are to be successful in encouraging the FDA to consistently review data related to all potential Duchenne therapies. If you have any experience on Translarna, or are waiting for access to Translarna, please share your experience with us so that we can translate your story into rigorous data that can be shared with FDA.
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Catabasis Quarterly Update - October 2016

Catabasis Quarterly Update - October 2016 | Duchenne Muscular Dystrophy Research | Scoop.it
ParentProjectMD's insight:
Catabasis provides a quarterly update on their MoveDMD trial with edasalonexent (CAT-1004) in Duchenne, highlighting the completion of enrollment for Part B of the MoveDMD trial and other recent news and events. We appreciate these updates and are proud to have a been an early supporter of this promising therapy.
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PTC Therapeutics Provides Regulatory Update on Translarna™

PTC Therapeutics Provides Regulatory Update on Translarna™ | Duchenne Muscular Dystrophy Research | Scoop.it
PPMD is disappointed to learn that the FDA denied PTC Therapeutics' first appeal of the refuse to file letter issued in February. As we understand it, this…
ParentProjectMD's insight:
PPMD is disappointed to learn that the FDA denied PTC Therapeutics' first appeal of the refuse to file letter issued in February. As we understand it, this is a process and may involve additional appeals. PPMD will be meeting with PTC to better understand the communications that transpired between the agency and the company and how we as a community can support this process moving forward.
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Catabasis Completes Target Enrollment for Part B of the MoveDMD® Trial, a Phase 2 Trial of Edasalonexent (CAT-1004) for the Potential Treatment of Duchenne Muscular Dystrophy

Catabasis Completes Target Enrollment for Part B of the MoveDMD® Trial, a Phase 2 Trial of Edasalonexent (CAT-1004) for the Potential Treatment of Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
Catabasis announced that target enrollment of 30 patients has been reached for Part B of the MoveDMD trial, a 12-week trial to assess the safety and efficacy…
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