PTC Therapeutics, Inc., today announced that it will make Translarna available for siblings of patients in certain open label PTC clinical trials for nonsense mutation Duchenne muscular dystrophy (nmDMD). Translarna will be made available to these siblings in advance of commercial availability in their regions, after a treating physician and the patient or guardian make a clinical decision to use Translarna, and consistent with any other applicable regulatory requirements.
PTC Therapeutics today announced that it will make Translarna available for siblings of patients in certain open label PTC clinical trials for nonsense mutation Duchenne in advance of commercial availability in their regions.
“Families of children with Duchenne have wanted companies to be responsive in the case of siblings. With this program, PTC, once again, demonstrates itself as a champion for the Duchenne community.”
A Northbrook company said it has won fast-track status for approval of a drug that could become the first in the United States to treat Duchenne muscular dystrophy , a fatal muscle disease found most often in boys.
Sarepta Therapeutics Inc. today announced that it has initiated dosing of SRP-4053 in its first human trial, a Phase I/II study in Duchenne muscular dystrophy (DMD). This multiple-dose study will assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in DMD patients with genotypes amenable to exon-53 skipping.
Sarepta Therapeutics Reports Long-Term Outcomes through 168 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy
“PPMD’s benefit-risk pilot data demonstrated that parents’ highest priority is to slow DMD progression. With this priority in mind, these Phase II data have exceeded our expectations” said Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), a nonprofit organization focused on finding a cure for Duchenne muscular dystrophy. “In practical terms, continued ambulation and preservation of respiratory function have immense benefit for individuals with Duchenne. It is important to keep in mind, that individuals participating in this study lived with Duchenne for over 9 years on average prior to receiving eteplirsen, an age when the pathological process that occurs based on the absence of dystrophin is typically well underway. Understanding this, in the eyes of the community these Phase II data are significant and represent an important step toward changing the landscape.”
PTC Therapeutics, Inc. today announced that it has commenced a rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Translarna™ for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). A rolling submission allows completed portions of the application to be submitted and reviewed by the FDA on an ongoing basis. PTC expects to finalize the application in the fourth quarter of 2015 following the completion of the ACT DMD confirmatory Phase 3 clinical trial.
We are excited that PTC will begin their rolling NDA submission to the FDA for Translarna! There is so much hope for 2015 in this community. Thank you to ALL of the companies that remain dedicated to our mission – end Duchenne.
Parent Project Muscular Dystrophy (PPMD) announced today a collaboration with Catabasis Pharmaceuticals, Inc. (Catabasis) to assist in a Phase 2 clinical tri…
We are excited to announce a collaboration with Catabasis Pharmaceuticals, Inc. to assist in a Phase 2 clinical trial of its CAT-1004 product candidate for the treatment of patients with Duchenne. As part of this collaboration, PPMD will provide funding to support participant travel. The trial is expected to begin in the first half of 2015. Click below to learn more about CAT-1004.
Advancing diagnosis, care and treatment for people with neuromuscular diseases around the world.
You may have seen the recent announcement from Phrixus about the availability of Carmaseal(TM) in the European Union (http://bit.ly/15SF1GN). UPPMD and TREAT-NMD have released a comment that provides more information about this topic.
PPMD is proud to announce a collaboration with Santhera Pharmaceuticals on a benefit/risk study that will focus specifically on patient and caregiver preferen…
We are proud to announce a collaboration with Santhera Pharmaceuticals on a new benefit/risk study that will focus specifically on patient and caregiver preferences regarding pulmonary therapies in the disease, and will be based on data from Santhera’s successful phase III clinical trial of idebenone.
PPMD will be reaching out to the community in the coming months to participate in this new study. Both patients and caregivers will be able to participate, even if you participated in PPMD’s previous benefit/risk study. We hope you’ll add your experiences so that we can continue to provide the FDA with as much information as possible in order to accelerate the decision-making process!
DuchenneConnect serves as a central hub linking the resources and needs of the Duchenne and Becker muscular dystrophy community: young men with Duchenne; their families and caregivers; and the provider community: clinical care providers, policymakers, industry professionals and the medical research fields.âWe offer registered members resources to: assist with early, appropriate and least invasive diagnosis; explore the benefits and limitations of genetic testing; offer access to resources and services, including care and treatment; and assist in understanding and development of new treatment trials.
"This capital raise will also fuel a clinical development program in our recently announced program to treat Duchenne Muscular Dystrophy (DMD) associated cardiomyopathy with CDCs, which we hope to be in the clinic in 2015. "
In a new study led by Dr. Subha Raman of The Ohio State University Wexner Medical Center, researchers were able to dramatically slow the rate of heart damage…
Early use of available heart failure drugs slows the progressive decline in heart function before cardiac symptoms are apparent in boys and young men with Duchenne, according to a new study published online by The Lancet Neurology. Thank you for helping PPMD fund this promising research!
Summit announces that it has received approval from the UK Medicines and Healthcare products Regulatory Agency and the Ethics Review Committee to initiate a Phase 1b modified diet trial of SMT C1100. SMT C1100 is an oral small molecule utrophin modulator in development for the potential treatment of all patients with DMD, regardless of the underlying dystrophin fault causing the disease. This new trial aims to increase the plasma levels of SMT C1100 by providing patients with specific dietary guidance intended to improve drug absorption. The trial will also evaluate the potential impact that SMT C1100 is having on enzyme markers of muscle health. Top-line data from the Phase 1b modified diet trial are expected to be reported in mid-2015.
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.
SOUTH PLAINFIELD, N.J., Dec. 3, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Translarna™ (ataluren) is now commercially available to patients in Germany, the first country to launch in the European Union (EU), with first shipments expected to begin this week.
"The launch of Translarna in Germany marks another exciting milestone for the DMD community," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "Through our reimbursed early access programs and commercial efforts, we are working hard to bring Translarna to those who may benefit from this drug. We remain committed to bringing Translarna to patients in Europe, and around the world, as quickly as possible. We know every day counts for the patients and their families."
The launch follows the European Commission marketing authorization, which was received in August 2014 for the use of Translarna to treat ambulatory patients with nonsense mutation Duchenne muscular dystrophy who are 5 years and older. A confirmatory Phase 3 clinical trial of Translarna, ACT DMD, in nonsense mutation Duchenne muscular dystrophy patients completed enrollment in September 2014. Top-line data is expected in the fourth quarter of 2015. Currently, Translarna is not approved for use in the United States.
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