The results of the Phase 2 trial into the drug, Vosoritide, that could reduce the health complications of children with Achondroplasia was published in the New England Journal of Medicine on Tuesday 18th June 2019.
Achondroplasia is a genetic bone disorder and is the most common of the conditions causing dwarfism with around 70% of people with dwarfism having this condition. The vast majority of children born with achondroplasia, around 80%, will have average-height parents and the genetic condition will have occurred spontaneously.
People with dwarfism live normal lives; working as doctors, lawyers, accountants, teachers and shop assistants; enjoying and taking part in the same hobbies and leisure activities as the rest of society; living in families with people of average height or short stature as their partners, husbands, wives, brothers, sisters and children.
A number of children and adults, with Achondroplasia, experience health complications as a result of their condition. These include; spinal cord compression, spinal curvature, bowed legs, joint pains and problems with the inner ear.
As a support charity we are passionate about changing society for the better, so that people with dwarfism can have the same opportunities as everyone else. Our purpose is to ensure that all people who are affected by dwarfism are equal in society. We would therefore support any medical advancements that reduce the health complications of people with achondroplasia, avoid surgery, remove chronic pain and allow people to live every aspect of their lives to the fullest.
RGAUK works closely with our Medical Advisory Board who are following the research and work being carried out on Vosoritide. Doctor Melita Irving from Guy’s and St Thomas’ NHS Foundation Trust is a member of the Medical Advisory Board and conducted part of the Phase 2 trials. RGAUK supports Guy’s and St Thomas’ NHS Foundation Trust and the drug trials and are delighted that the trial has, so far, shown a potential to reduce the health complications of people with Achondroplasia.
There has been some discussion around the additional height gained as an outcome of the trial. Whilst this may help individuals when it comes to reaching things more comfortably throughout the day, RGAUK do not view this increased height as an important outcome, and if it is shown that this is the only benefit of Vosoritide and there is no reduction in the health complications, RGAUK would no longer support the development of the drug.
It’s been a little while but we’re back with a fantastic new Role Model in the Dwarfism Community interview! This time, we had the privilege of speaking with Mia Ives-Rublee, a civil rights activist. Check out what she had to say. Read More
People with dwarfism are often misrepresented in media and popular culture. Yet our own voices are rarely heard. That’s why we started our Role Models in the Dwarfism Community series – to showcase figureheads in our own communities. Today, we spoke with Cara Reedy, writer, actor, producer and director.Read More
People with dwarfism are often misrepresented in popular media and culture. Yet our own voices are rarely heard. That’s why we embarked upon the RGA Dwarfism Role Models project – to amplify the voices of figureheads in our communities. For this, our latest instalment, we spoke with Joe Stramondo, Assistant Professor of Philosophy at San Diego State University.Read More
We’re back – with yet another great installment in our series of interviews with role models from the dwarfism community. This time we spoke to Vilissa Thompson – social worker, writer, and self-confessed “badass”. Check out what she had to say.Read More
Happy New Year! We’re back with a new installment in our series of interviews with role models in dwarfism communities. We spoke to artist and academic, Dr Debra Keenahan.Read More
We’re back with another great interview in our role models in the dwarfism community. This time, we spoke to Dr Will Christian – a consultant in paediatric emergency medicine – who had some excellent advice for us all. Read More
Continuing our popular series of interviews with role models in the dwarfism community we spoke with John Young, teacher, coach, athlete and marathon runner. Check out what he had to say. Read More
My name is Jacob. I always introduce myself as a twenty-something software developer, gamer; who has a Christmas cracker sense of humour; a slight affinity for caffeine; and who just happens to have Achondroplasia.
I saw the RGA was running the #SpreadTheWord campaign – asking libraries and schools to stock books that show a positive image of dwarfism. I wanted to take part and contacted my old primary school to see if they’d welcome the books into their library. The current generation there would not have grown up with any real-life representation of dwarfism. They accepted my request and wanted me to revisit the school to deliver a presentation.Read More
Wowzers! We’ll be hosting our annual Mega Weekend event between October 26th and 29th 2017 at the Woodland Grange hotel – of which we have exclusive use – in Leamington Spa, Warwickshire.
Three full days in which people with dwarfism and their families can attend a range of interactive workshops, informative talks, and fun activities, including:Read More