Duchenne Muscular Dystrophy Research
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Prosensa Wins Scrip 'Biotech Company of the Year' 2014 Award (NASDAQ:RNA)

Prosensa Wins Scrip 'Biotech Company of the Year' 2014 Award (NASDAQ:RNA) | Duchenne Muscular Dystrophy Research | Scoop.it

Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with a high unmet medical need, is delighted to have won the Scrip 'Biotech Company of the Year' Award for 2014, announced last week in London, UK.

The prestigious Scrip Awards provide an opportunity to acknowledge and applaud the highest achievers across the biotechnology and pharmaceutical industry, recognizing both corporate and individual accomplishments. The award for 'Biotech Company of the Year' is given to the company which has demonstrated the greatest achievements for the year.

Hans Schikan, CEO of Prosensa, said, "Winning Scrip's 'Biotech Company of the Year' Award is a great honor and a testament to the dedication and hard work of the Prosensa team.  Our drive to succeed is fueled by patients and their families, who continue to inspire us to make innovative therapies available as quickly and efficiently as possible."

The other five finalists were: Ablynx; Horizon Discovery; Isis Pharmaceuticals; Mesoblast; and Silence Therapeutics.

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Eloxx Pharmaceuticals Announces First Patient Dosed in Phase 1b Clinical Trial

Lead molecule targets genetic nonsense mutation in rare diseasesDose escalating study to enroll 36 subjectsData expected in the fourth quarter of 2018SA
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Sarepta Therapeutics Announces FDA Clearance of IND for the Company’s PPMO Exon 51 Candidate, SRP-5051 | Sarepta Therapeutics

Sarepta Therapeutics Announces FDA Clearance of IND for the Company’s PPMO Exon 51 Candidate, SRP-5051 | Sarepta Therapeutics | Duchenne Muscular Dystrophy Research | Scoop.it
The Investor Relations website contains information about Sarepta Therapeutics's business for stockholders, potential investors, and financial analysts.
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Sarepta Therapeutics Signs Exclusive Global Collaboration with Duke University for Gene Editing CRISPR/Cas9 Technology to Develop New Treatments for Duchenne Muscular Dystrophy (DMD) | Sarepta Ther...

Sarepta Therapeutics Signs Exclusive Global Collaboration with Duke University for Gene Editing CRISPR/Cas9 Technology to Develop New Treatments for Duchenne Muscular Dystrophy (DMD) | Sarepta Ther... | Duchenne Muscular Dystrophy Research | Scoop.it
The Investor Relations website contains information about Sarepta Therapeutics's business for stockholders, potential investors, and financial analysts.
ParentProjectMD's insight:
PPMD is excited to see today’s news from Sarepta Therapeutics announcing an exclusive global collaboration with Duke University for Gene Editing CRISPR/Cas9 Technology to develop new treatments for Duchenne. If you attended PPMD’s Every Single One Tour stop at Duke University late last year, you heard Dr. Charles A. Gersbach talk about the promising gene editing work his lab is doing. Thank you to Sarepta and the team at Duke for your innovation. PPMD will continue to invest in technology like CRISPR/Cas9 as part of our Gene Therapy Initiative.
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[Webinar] FibroGen Update on Pamrevlumab & Enrolling Non-Ambulatory Study - October 2017

PPMD and FibroGen hosted a webinar on October 25, 2017 to hear a community update on FibroGen’s development of Pamrevlumab and currently enrollin
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2017 World Muscle Society: Highlighting Progress in Duchenne

2017 World Muscle Society: Highlighting Progress in Duchenne | Duchenne Muscular Dystrophy Research | Scoop.it
“WMS - What’s the buzz?”
 
It has been many years since I’ve had the opportunity to attend the World Muscle Society meeting and wow how things have changed!…
ParentProjectMD's insight:
The World Muscle Society meeting is the annual gathering that brings together the world’s top neuromuscular clinicians, academics, research scientists, and industry leaders from across the globe. Topics presented here feature breakthroughs and highlights in diseases across the neuromuscular spectrum. Over the next few days, PPMD will be sharing highlights from Duchenne presentations and posters, as well as the announcements that are being released from companies in our Duchenne space in conjunction with the meeting. It is incredible to be part of this extraordinary gathering of partners who are excited to be working with our Duchenne community and like PPMD, are eager to accelerate therapy development efforts.
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[Webinar] MoveDMD Trial Open-Label Extension Results

[Webinar] MoveDMD Trial Open-Label Extension Results | Duchenne Muscular Dystrophy Research | Scoop.it
Please join Parent Project Muscular Dystrophy and Catabasis Pharmaceuticals as we host a webinar Wednesday, October 11 at 1:00 PM EDT to hear a community upd…
ParentProjectMD's insight:
Join PPMD and Catabasis Pharmaceuticals as we host a webinar Wednesday, October 11 at 1 PM EDT to hear a community update on the results from the open-label extension of the MoveDMD trial.
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Webinar Q&A: Gene Therapy Part 2 – Nationwide Children's Hospital

Webinar Q&A: Gene Therapy Part 2 – Nationwide Children's Hospital | Duchenne Muscular Dystrophy Research | Scoop.it
This fall, PPMD is presenting a gene therapy webinar series with companies and institutions who are developing therapies for Duchenne that are commonly refer…
ParentProjectMD's insight:
Dr. Jerry Mendell of Nationwide Children’s Hospital recently joined PPMD for a webinar to discuss his upcoming gene therapy trial, including trial design, inclusion/exclusion criteria, and timelines. There were some great questions asked, but unfortunately we were not able to get to all of them during the live webinar. The remaining questions have been addressed in our blog.
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Synpromics Announces Gene Therapy Research Partnership with Solid Biosciences

Synpromics Announces Gene Therapy Research Partnership with Solid Biosciences | Duchenne Muscular Dystrophy Research | Scoop.it
Synpromics Announces Gene Therapy Research Partnership with Solid Biosciences
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Phrixus Pharmaceuticals announces Phase 2 clinical trial of Poloxamer 188 NF in non-ambulatory patients with Duchenne muscular dystrophy

Phrixus Pharmaceuticals announces Phase 2 clinical trial of Poloxamer 188 NF in non-ambulatory patients with Duchenne muscular dystrophy | Duchenne Muscular Dystrophy Research | Scoop.it
Phrixus to test Carmeseal-MD™ (P-188 NF) at Cincinnati Children’s Hospital in a single-site, open-label trial against respiratory and cardiac end points
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First Patients Enrolled in UK’s Early Access to Medicines Scheme for Santhera’s Raxone® in Duchenne Muscular Dystrophy (DMD)

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First Patients Enrolled in UK’s Early Access to Medicines Scheme for Santhera’s Raxone® in Duchenne Muscular Dystrophy (DMD)
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FibroGen soars as idiopathic pulmonary fibrosis data paint pamrevlumab as a blockbuster, spark talk of big partnership | FierceBiotech

FibroGen soars as idiopathic pulmonary fibrosis data paint pamrevlumab as a blockbuster, spark talk of big partnership | FierceBiotech | Duchenne Muscular Dystrophy Research | Scoop.it
FibroGen has convinced investors it has another potential blockbuster on its hands. Shares in the West Coast biotech shot up 60% after it posted top-line phase 2 pamrevlumab data that suggest the anti-connective tissue growth factor (CTGF) antibody can muscle in on a market fought over by Boehringer Ingelheim and Roche.
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Sarepta Therapeutics and Clinigen Launch a Managed Access Program to Treat Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Sarepta Therapeutics and Clinigen Launch a Managed Access Program to Treat Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy Research | Scoop.it
The MAP will be available to a limited number of patients who meet pre-specified medical criteria and can secure funding
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Summit Completes Initial 24 Weeks of Dosing of Ezutromid in Patients with DMD in PhaseOut DMD Clinical Trial

Summit Completes Initial 24 Weeks of Dosing of Ezutromid in Patients with DMD in PhaseOut DMD Clinical Trial | Duchenne Muscular Dystrophy Research | Scoop.it
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Accelerating the Clinical Trial Process in Duchenne

Accelerating the Clinical Trial Process in Duchenne | Duchenne Muscular Dystrophy Research | Scoop.it
Lengthy trials with variable results, based on endpoints such as the 6-minute walk test that can limit trial enrollment to ambulant individuals with Duchenne…
ParentProjectMD's insight:
Lengthy trials with variable results, based on endpoints such as the 6-minute walk test that can limit trial enrollment to ambulant individuals with Duchenne – this is today’s reality for clinical trials in this disease. But progress is being made and PPMD is proud of our role in changing the clinical trial process. Learn more about current progress that will one day make clinical trials shorter and better for participants.
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Eloxx Pharmaceuticals Announces Expansion of Intellectual Property Estate for ELX-02 with Newly Granted Patent in Europe

SAN DIEGO and TEL AVIV, Israel and BOSTON, Oct. 16, 2017 /PRNewswire/ -- Sevion Therapeutics, Inc. (OTCQB:SVON) and Eloxx Pharmaceuticals Ltd.,
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[Webinar] MoveDMD Trial Open-Label Extension Results - October 2017

PPMD and Catabasis Pharmaceuticals hosted a webinar on October 11, 2017 to hear a community update on the results from the open-label extension of th
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Catabasis Reports Positive Results from Open-Label Extension of MoveDMD Trial with Edasalonexent

Catabasis Reports Positive Results from Open-Label Extension of MoveDMD Trial with Edasalonexent | Duchenne Muscular Dystrophy Research | Scoop.it
Catabasis Pharmaceuticals, Inc. today reported new positive efficacy results following 24 and 36 weeks of treatment with 100 mg/kg/day oral edasalonexent in th…
ParentProjectMD's insight:
Catabasis Pharmaceuticals today reported new positive efficacy results following 24 and 36 weeks of treatment with 100 mg/kg/day oral edasalonexent in the MoveDMD Phase 2 trial open-label extension. PPMD is excited to see positive effects on muscle function with edasalonexent. Please join us as we host a webinar with Catabasis on Wednesday, October 11 at 1:00 PM EDT to hear a community update on these results. More information can be found in our blog.
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Letter from PTC Therapeutics to the Community Following FDA Ad Comm

Letter from PTC Therapeutics to the Community Following FDA Ad Comm | Duchenne Muscular Dystrophy Research | Scoop.it
PTC Therapeutics President and CEO, Stuart Peltz, Ph.D has shared a letter with the Duchenne community following last week's Advisory Committee Meeting at the…
ParentProjectMD's insight:
PTC Therapeutics President and CEO, Stuart Peltz, Ph.D has shared a letter with the Duchenne community following last week's Advisory Committee Meeting at the FDA for ataluren. PTC hopes FDA will consider all the information heard at the Advisory Committee meeting, including the experiences that were shared as they complete their review. The target date for the FDA to complete their review (referred to as the PDUFA date) is October 24, 2017. PPMD will continue to bring you updates as they become available.
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Capricor Therapeutics Advances Clinical Development Program in Duchenne Muscular Dystrophy with Naming of Principal Investigator

Capricor Therapeutics Advances Clinical Development Program in Duchenne Muscular Dystrophy with Naming of Principal Investigator | Duchenne Muscular Dystrophy Research | Scoop.it
LOS ANGELES, Sept. 26, 2017 /PRNewswire/ -- Capricor Therapeutics Advances Clinical Development Program in Duchenne Muscular Dystrophy with Namin
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[Webinar] PART 2: Gene Therapy for Duchenne - Nationwide Children's Hospital - September 2017

On September 6, 2017 Dr. Jerry Mendell of Nationwide Children’s Hospital joined PPMD for a webinar to discuss his upcoming gene therapy trial, includin
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Phrixus Pharmaceuticals announces the first patient with Duchenne muscular dystrophy (DMD) to meet the 15-month mark on Carmeseal-MD™ (Poloxamer 188 NF)

Phrixus Pharmaceuticals announces the first patient with Duchenne muscular dystrophy (DMD) to meet the 15-month mark on Carmeseal-MD™ (Poloxamer 188 NF) | Duchenne Muscular Dystrophy Research | Scoop.it
- First patient to be treated with Carmeseal-MD (P-188 NF) outside of the United States through Phrixus’s Expanded Access Program with Ethicor Pharma Ltd. -
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Mitobridge’s Novel Treatment Approach for Duchenne Muscular Dystrophy Advances into Clinical Development

Mitobridge’s Novel Treatment Approach for Duchenne Muscular Dystrophy Advances into Clinical Development | Duchenne Muscular Dystrophy Research | Scoop.it
Mitobridge, Inc., a pioneer in the discovery and development of products that improve mitochondrial function, today announces a key milestone with the
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Sarepta Therapeutics Announces its Partner, Genethon, Published New Micro-Dystrophin Gene Therapy Data in Nature Communications

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