21 Mar
  • By cure.duchenne
  • Cause in

SUMMIT OUTLINES CLINICAL DEVELOPMENT PLANS FOR UTROPHIN MODULATOR PROGRAMME FOR DUCHENNE MUSCULAR DYSTROPHY

• First patient clinical trials of SMT C1100 expected to start H2 2013 •Summit to showcase programme at international scientific conference Oxford, UK, 21 March 2013 – Summit (AIM: SUMM), a drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy (‘DMD’) and C. difficile infections, today outlines its future plans for the continued […]

READ MORE
04 Mar
  • By cure.duchenne
  • Cause in

Drisapersen/GSK Update

Dear Debra, Since my last email you may have seen a recent story that appeared on an investment tips website regarding a presentation on drisapersen made at the Duchenne Parent Project Onlus meeting on 24th February 2013 in Rome, Italy. We appreciate that the story may raise questions for you, so I wanted to write and […]

READ MORE
21 Feb
  • By cure.duchenne
  • Cause in

FDA Webinar on Accelerated Approval Summary

    CureDuchenne was honored to host a webinar today, in collaboration with PPMD and MDA to discuss accelerated approval for drugs for rare diseases.   Dr. Robert Temple, director of the Office of Medical Policy of FDA’s Center for Drug Evaluation and Research presented detailed information on the different options available to speed the approval of […]

READ MORE
29 Jan
  • By cure.duchenne
  • Cause in

Prosensa Achieves Orphan Drug Status on its Entire

  Leiden, The Netherlands – 29 January 2013 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, announced it has achieved orphan drug designation in the EU and the US for all of its compounds for the treatment of Duchenne muscular dystrophy (DMD). The European Medicines Agency […]

READ MORE
10 Jan
  • By cure.duchenne
  • Cause in

GSK Exon 51 Trial Update

GSK has sent out this update on their Duchenne trial.  The last patient has been recruited within the drisapersen US Phase II clinical study [www.clinicaltrials.gov NCT01462292]. This exploratory study aims to assess the safety, efficacy and pharmacokinetics of two doses of drisapersen in the treatment of ambulant boys with DMD who have a dystrophin gene […]

READ MORE
28 Dec
  • By cure.duchenne
  • Cause in

Cure Duchenne Supports Duchenne Duplication Mutation Research with Dr. Kevin Flanigan

  Exon duplication mutations have been an area of great interest to CureDuchenne, and last year, CureDuchenne funded research specifically directed toward these mutations in Duchenne.  Dr. Kevin Flanigan at Nationwide Children’s Hospital has successfully completed the first phase of his research (please see below). CureDuchenne will continue to fund his work in 2013. The three […]

READ MORE
13 Dec
  • By cure.duchenne
  • Cause in

Henri Termeer joins Prosensa as Strategic Advisor

 We are so pleased to see the high caliber of professionals joining in the fight to cure Duchenne. Henri Termeer served as Chairman, President and CEO of Genzyme Corporation for nearly three decades.  He created a system where patients with rare diseases were able to have access to very expensive drugs. He joins an incredible […]

READ MORE
12 Dec
  • By cure.duchenne
  • Cause in

Dantrolene: An Exon Skipping Enhancing Drug

Drs. Carrie Miceli and Stan Nelson at UCLA are developing an FDA approved drug that could boost the effectiveness of exon skipping drugs that are being developed and tested for Duchenne muscular dystrophy. Drs. Miceli and Nelson in collaboration with Dr. Melissa Spencer, have tested dantrolene in combination with morpholinos in the mdx mouse, the mouse model […]

READ MORE
11 Dec
  • By cure.duchenne
  • Cause in

nNOS: How Important is it in Duchenne?

nNOS has been in the news lately so we asked CureDuchenne’s chief scientific advisor, Dr. Mike Kelly to answer a few questions.  Question: what is nNOS and why do we need it? nNOS stands for neuronal nitric oxide synthase; its function is to produce nitric oxide and when needed, (in a simplistic view) help increase blood flow in […]

READ MORE
07 Dec
  • By cure.duchenne
  • Cause in

CureDuchenne – Clinical Trials – FDA

Following the long awaited news of Sarepta’s phase IIB results for eteplirsen and continued Phase III progress of GSK/Prosensa’s drisapersen, as well as the recent developments with PTC Therapeutics and Summit PLC among others, CureDuchenne took the initiative to reach out for patients and advocacy groups alike to begin a dialogue with the FDA and […]

READ MORE