Duchenne Muscular Dystrophy Research
95.4K views | +7 today
Follow
 
Scooped by ParentProjectMD
onto Duchenne Muscular Dystrophy Research
Scoop.it!

Prosensa Patient Group Communication April 2014

ParentProjectMD's insight:

The latest update from Prosensa regarding re-dosing drisapersen and the regulatory path forward.

more...
No comment yet.
Duchenne Muscular Dystrophy Research
Your new post is loading...
Your new post is loading...
Scooped by ParentProjectMD
Scoop.it!

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
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

FDA Grants Orphan Drug Designation to Mallinckrodt Development Product for Potential Treatment of Duchenne Muscular Dystrophy

STAINES-UPON-THAMES, United Kingdom, July 12, 2017 /PRNewswire/ -- FDA Grants Orphan Drug Designation to Mallinckrodt Development Product fo
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Sarepta Therapeutics and Genethon Announce a Gene Therapy Research Collaboration for the Treatment of Duchenne Muscular Dystrophy

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Pfizer Completes Enrollment of Phase 2 Study of Domagrozumab (PF- 06252616)in Duchenne Muscular Dystrophy

Empty description
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Santhera to Present Data and Host Symposium on Pulmonary Function in Duchenne Muscular Dystrophy at the American Thoracic Society 113th Annual Conference

Santhera Pharmaceuticals, a specialty pharmaceutical company focused on the development of innovative treatments for rare mitochondrial and neuromuscular diseases, announces that two posters with data from the positive phase III DELOS trial in patients with Duchenne muscular dystrophy (DMD) will be presented at the 113th American Thoracic Society (ATS) International Conference in Washington D.C.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

PTC Therapeutics to Discuss Launch Plans for Emflaza™ (Deflazacort) during Community Call on Monday, May 8th at 3:00 PM EDT

PTC Therapeutics to Discuss Launch Plans for Emflaza™ (Deflazacort) during Community Call on Monday, May 8th at 3:00 PM EDT | Duchenne Muscular Dystrophy Research | Scoop.it
PTC Therapeutics has released a letter to the community and will host a conference call for the Duchenne community to discuss the launch plans for EMFLAZA™ (d…
ParentProjectMD's insight:
PTC Therapeutics has released a letter to the community and will host a conference call for the Duchenne community to discuss the launch plans for EMFLAZA™ (deflazacort) on Monday, May 8, 2017 at 3:00 PM (ET). Call-in details are below. On a public call to investors earlier this morning, PTC announced that the price for EMFLAZA in the U.S. will start at $35,000 for a patient weighing 25kg (approximately 55lbs), and would fluctuate based on a patient’s dose and weight. PPMD is anxious to learn more details about how PTC arrived at this pricing structure, as well as access plans, on today’s call.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Catabasis Quarterly Update: April 2017

Catabasis Quarterly Update: April 2017 | Duchenne Muscular Dystrophy Research | Scoop.it
Catabasis provides a quarterly update on their MoveDMD trial with edasalonexent (CAT-1004) in Duchenne.
ParentProjectMD's insight:
Catabasis provides a quarterly update on their MoveDMD trial with edasalonexent (CAT-1004) in Duchenne, discussing more of the functional results from Part B of the trial and the current activities in the ongoing open-label extension (Part C). We appreciate these updates and are proud to have a been an early supporter of this promising therapy.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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…
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

[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
ParentProjectMD's insight:
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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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…
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

[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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Capricor Receives Rare Pediatric Disease Designation from FDA for CAP-1002 for Patients with Duchenne Muscular Dystrophy

Empty description
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Santhera's Raxone® Receives First Positive EAMS Scientific Opinion from UK's MHRA in Duchenne Muscular Dystrophy

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

[Webinar] ESSENCE: Sarepta’s Exon 45 and 53 Skipping Study - June 2017

PPMD and Sarepta Therapeutics hosted a webinar June 7, 2017 for an overview of Sarepta’s pivotal study investigating SRP-4045 and SRP-4053 for the treatmen
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

PTC Therapeutics Provides EMFLAZA™ (Deflazacort) FAQ Update

PTC Therapeutics has provided an update regarding EMFLAZA (see below). Please visit EMFLAZA.com for answers to some of the most frequently asked questions PTC…
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Santhera Provides Update on Timeline for Application of Raxone® in Duchenne Muscular Dystrophy in Europe

more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

Summit Completes Enrolment of PhaseOut DMD, a Phase 2 Clinical Trial of Ezutromid in Patients with DMD

Summit Completes Enrolment of PhaseOut DMD, a Phase 2 Clinical Trial of Ezutromid in Patients with DMD | Duchenne Muscular Dystrophy Research | Scoop.it
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

[Webinar] Givinostat in Duchenne - April 2017

Parent Project Muscular Dystrophy and Italfarmaco hosted a webinar on April 26, 2017 to discuss Italfarmaco's investigational drug Givinostat (a histon
ParentProjectMD's insight:
ICYMI, Italfarmaco joined us for a webinar last week to discuss the investigational drug Givinostat (a histone deacetylase inhibitor) and how it works in Duchenne. Our speakers presented a brief history of the development program, including pre-clinical results and preliminary results of the current Phase 2 clinical trial, study 43, which is ongoing in Italy, and answered questions from our community. If you missed it, you can now watch the recording of the webinar below.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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…
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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…
ParentProjectMD's insight:
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.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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
Empty description
ParentProjectMD's insight:
Bristol-Myers Squibb has provided information on their Anti-Myostatin Adnectin Program (BMS-986089) and announced their planned global study, CN001-016.
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

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
more...
No comment yet.
Scooped by ParentProjectMD
Scoop.it!

[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
more...
No comment yet.