Duchenne Muscular Dystrophy Research
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Capricor Therapeutics Announces Positive Six-Month Results from the Randomized Phase I/II HOPE Clinical Trial in Duchenne Muscular Dystrophy

Capricor Therapeutics Announces Positive Six-Month Results from the Randomized Phase I/II HOPE Clinical Trial in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
Capricor Therapeutics today announced positive top-line results from a safety and exploratory efficacy analysis of six-month data from the randomized 12-month…
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PTC Therapeutics Completes Acquisition of Emflaza™ for the Treatment of Duchenne in the U.S.

PTC Therapeutics Completes Acquisition of Emflaza™ for the Treatment of Duchenne in the U.S. | Duchenne Muscular Dystrophy Research | Scoop.it
Today, PTC Therapeutics announced that they have completed their acquisition of Emflaza™ (deflazacort) ahead of schedule, following early conclusion of the ant…
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Today, PTC Therapeutics announced that they have completed their acquisition of Emflaza™ (deflazacort) ahead of schedule, following early conclusion of the anti-trust review. PTC will be hosting a call with the Duchenne community the week of May 8 to provide details on the plan for the launch of Emflaza. We look forward to learning more from PTC in the coming weeks and will keep you posted on call-in details.
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Bristol-Myers Squibb (BMS) Provides Update on Anti-Myostatin Adnectin Program

Bristol-Myers Squibb (BMS) Provides Update on Anti-Myostatin Adnectin Program | Duchenne Muscular Dystrophy Research | Scoop.it
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Bristol-Myers Squibb has provided information on their Anti-Myostatin Adnectin Program (BMS-986089) and announced their planned global study, CN001-016.
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Solid Biosciences Raises up to $50 Million in Series C Financing

Solid Biosciences Raises up to $50 Million in Series C Financing | Duchenne Muscular Dystrophy Research | Scoop.it
Solid Biosciences announced today that it has completed the initial closing of its $50 Million Series C financing, which the company will use to progr
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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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Gene-editing alternative corrects Duchenne muscular dystrophy: April 2017 News Release - UT Southwestern, Dallas, TX

Gene-editing alternative corrects Duchenne muscular dystrophy: April 2017 News Release - UT Southwestern, Dallas, TX | Duchenne Muscular Dystrophy Research | Scoop.it
Using the new gene-editing enzyme CRISPR-Cpf1, researchers at UT Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells and mice in the lab.
ParentProjectMD's insight:
PPMD is excited to see promising news regarding the potential of CRISPR gene editing technology in Duchenne. These findings by Dr. Eric Olson and the Department of Molecular Biology at UT Southwestern Medical Center are the first to show the efficiency of Cpf1-mediated correction of genetic mutations in human cells and an animal disease model – providing us with a promising new tool in the CRISPR toolbox. Thanks to your support, PPMD was able to award a $250,000 grant to Dr. Olson and his lab earlier this year, as part of PPMD’s gene transfer initiative.
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Bristol-Myers Squibb Enters into Agreement with Roche to License Anti-Myostatin Compound

Bristol-Myers Squibb Enters into Agreement with Roche to License Anti-Myostatin Compound | Duchenne Muscular Dystrophy Research | Scoop.it
Bristol-Myers Squibb (BMS) today announced that it has entered into an agreement to license BMS-986089, an anti-myostatin adnectin in development for Duchenne…
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[Webinar] Akashi Provides Update on HT-100 - March 2017

Parent Project Muscular Dystrophy and Akashi Therapeutics hosted a webinar on March 27, 2017 to discuss HT-100 and next steps for the clinical program
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Parent Project Muscular Dystrophy and Akashi Therapeutics hosted a webinar on March 27, 2017 to discuss HT-100 and next steps for the clinical program. Akashi CEO Marc Blaustein presented data from the previous clinical program of HT-100, discussed FDA’s recent decision to allow the clinical development of HT-100 to resume, and answered questions from the community.
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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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