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News & Events

The Challenges of Daily Life - Duchenne Muscular Dystrophy

2 August 2017

Aside from progressively weakening muscles, Duchenne Muscular Dystrophy (DMD) is also responsible for the stiffening and constriction of tendons, ligaments, and other connective tissues, rendering them inflexible and resulting in limited movements.

Certain activities that require intense motion such as playing sports or simply walking, running, and jogging become extremely difficult, which is why people with muscular dystrophy often find themselves in need of the aid of certain equipment such as strollers, walkers, braces, crutches, and wheelchairs to be capable of moving around.

To read more: https://www.findatopdoc.com/Healthy-Living/Living-...

Clinical Trial Shows Ataluren Can Slow Down the Progression of Duchenne Muscular Dystrophy

20 July 2017

The results of an international clinical trial show that a drug therapy for a severe type of muscular dystrophy holds promise for a subgroup of patients with Duchenne muscular dystrophy. The trial was led in part by Craig McDonald, a UC Davis School of medicine expert.

“What’s very encouraging about these findings is that a treatment using ataluren for certain individuals with Duchenne muscular dystrophy may prompt a slowing or stabilizing of the disease progression and motor function that can be very meaningful to patients and provide them with invaluable, quality-of-life benefits,” McDonald said.

To read more: http://www.davisenterprise.com/local-news/clinical...

Muscular Dystrophy - The Three Best Treatment Methods

13 July 2017

There is no definite cure for Muscular Dystrophy but there are ways to manage the condition such as physical therapy, doctor prescribed medication and surgery.

To read more: https://www.findatopdoc.com/Healthy-Living/The-Thr...

Muscular Dystrophy - Telling a Child the Truth about Their Condition

13 July 2017

Muscular Dystrophy (MD) is a category of muscle diseases that cause progressive weakness and loss of muscle mass. 

MD is either inherited or can be due to a genetic mutation. 

Kids with MD usually follow a normal pattern of growth development in the first few years of their lives, but the signs and symptoms of MD gradually take place as time goes by.

Being honest with your child is the best way for them to understand why they are experiencing such things.

To read more: https://www.findatopdoc.com/Healthy-Living/Muscula...

How to Explain Muscular Dystrophy to Family and Friends

9 July 2017

Unless personally affected by muscular dystrophy, no one can fully come close to understanding it or understanding what it is like to deal with. Explaining muscular dystrophy to your friends and family will take time, and you might need to remind some people of what you’re going through.

Explaining muscular dystrophy to your friends and family will play a vital role in your acceptance of your current situation.

To read more: https://www.findatopdoc.com/Healthy-Living/Explain...

Local Business BarItalia Leichhardt Raises funds for HopeforOllie

24 December 2016

Local business support means so much! BarItalia in Leichhardt has been a regular HopeforOllie supporter since June 2016. 

Here is a video link of the Christmas Hamper kindly created by the owners, Con and Sarina, to raise funds for DMD clinical research https://www.facebook.com/BarItaliaLeichhardt/video...

Thank you so much x

Duchenne Children’s Balance Deteriorates as Disease Progresses, Study Finds

16 March 2017

The balance of children with Duchenne muscular dystrophy who are moving becomes worse as the disease progresses, suggesting that balanced-focused interventions may improve the children’s ability to handle everyday tasks.

To read more: https://musculardystrophynews.com/2017/03/15/study...

Vamorolone (VBP 15) Gets Fast Track Designation for Duchenne Muscular Dystrophy

25 March 2107

The FDA has granted Fast Track Designation for vamorolone (VBP15) for the treatment of patients with Duchenne muscular dystrophy to ReveraGen BioPharma Inc.

The VISION-DMD clinical trial program for vamorolone is currently enrolling boys with DMD into clinical trials in US, Canada, Australia, Sweden, UK, and Israel (open label Phase 2a, Phase 2a extension, and Long-term extension studies). Phase 2b trials are expected to initiate enrolment later this year.

To read more : http://www.raredr.com/news/vamorolone-fast-track

HopeforOllie Gingerbread Fundraiser 2016 a HUGE success!

18 December 2016

A big thank you to Louisa and Kylie from GreenBubbleGroup for raising over $2135 for HopeforOllie during the 3rd annual Gingerbread House Fundraiser. I'm so appreciative to the support shown by the local community.

All money raised was donated to DMD clinical research at The Children's Hospital at Westmead.

European Medicines Agency Renews Conditional Marketing Authorisation of Translarna

12 November 2016

CHMP Recommends Renewal of Translarna's™ Marketing Authorization for Nonsense Mutation Duchenne Muscular Dystrophy Based on Continued Positive Benefit-Risk Assessment

--PTC to conduct a post-authorization clinical trial --

SOUTH PLAINFIELD, N.J., Nov. 11, 2016 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients five years and older. In connection with the renewal, the marketing authorization will include a specific obligation to conduct an additional long-term post-authorization trial.

Read more: http://ir.ptcbio.com/releaseDetail.cfm?ReleaseID=9...