Duchenne Muscular Dystrophy Research
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Akashi Therapeutics Receives FDA Clearance to Resume HT-100 Clinical Development

Akashi Therapeutics Receives FDA Clearance to Resume HT-100 Clinical Development | Duchenne Muscular Dystrophy Research | Scoop.it
PPMD is pleased to learn that the FDA has completed its review and concluded that Akashi Therapeutics may resume clinical development of HT-100 (delayed-releas…
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[Webinar] MoveDMD Trial: Catabasis Provides Update - February 2017

The Catabasis team spoke to our community on February 2, 2017 to discuss what they learned from this latest trial and what comes next in the MoveDMD program.
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[Webinar] Summit's Utrophin Modulation Program - January 2017

On January 25, 2017, PPMD and Summit hosted an educational webinar on utrophin modulation, an approach for Duchenne that has the potential to treat al
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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…
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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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[Webinar] NS Pharma's Exon 53 Skipping Program: Trial Recruitment & Update - February 2017

PPMD and NS Pharma hosted a webinar on February 22, 2017 to provide an update on trial recruitment for NS Pharma's Exon 53 Skipping Program. Dr. Clemens an
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[Webinar] Mission DMD: FibroGen’s Anti-Fibrosis Program - February 2017

On February 1, 2017, PPMD and FibroGen hosted a webinar to discuss FibroGen’s investigational drug Pamrevlumab (FG-3019), along with FibroGen’s Mission DM
ParentProjectMD's insight:
ICYMI, FibroGen joined us earlier this week for a webinar discussion on the company’s investigational drug Pamrevlumab (FG-3019), which is an anti-CTGF monoclonal antibody, along with FibroGen’s Mission DMD program that is currently being conducted to investigate Pamrevlumab in Duchenne. Our speakers provided an overview of the rationale for evaluating Pamrevlumab in Duchenne patients, as well as a description of the recent protocol amendment for this ongoing clinical trial in non-ambulatory boys with Duchenne. 
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Santhera Reports Preliminary Key Financial Figures for 2016 and Provides Corporate Update

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[Webinar] Vamorolone (VBP15) Clinical Trials in Duchenne - January 2017

On January 18, 2017, PPMD was joined by ReveraGen BioPharma for a webinar discussion on vamorolone (VBP15) clinical trials in Duchenne. Dr. Eric Hoffma
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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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