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Healx raise funding to repurpose drugs for rare diseases

Healx raise funding to repurpose drugs for rare diseases

Congratulations to Cambridge start-up, Healx, co-founded by founder and trustee of CRDN, as it raises $10m to help repurpose drugs for rare diseases.

Forbes and the Telegraph today report that the co-founders of Healx, Tim Guilliams – founder and trustee of CRDN, and British inventor of Viagra – Dr David Brown, have raised funding of $10m for their Cambridge start-up that uses artificial intelligence (AI) technology to find medicines to treat some of the world’s rarest diseases.

The Telegraph states “Dr David Brown, the scientist who developed the blockbuster treatment for erectile dysfunction for Pfizer, is the co-founder of Healx, a UK medical tech startup that uses machine learning to find treatments for 7,000 rare conditions that do not currently have an approved method of treatment”.

 

It goes on to explain why Healx believe that the lengthy traditional drug discovery process isn’t economic for rare diseases and how they hope to accelerate the time to drug discovery and reduce costs through their AI technology driven systems which search for drugs that can be repurposed. 

Dr Brown’s work with Viagra, which was developed originally to treat heart patients, proved that drugs intended to help one condition can sometimes be adapted to treat others. To read the full article click here.

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© Cambridge Rare Disease Network 2018
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Reg'd Charity Number: 1166365
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CRDN Summit 2017 – The Film

CRDN Summit 2017 – The Film

We collaborated with Lucia Tambini,  film maker producing films for charities including Oxfam, Tourettes Action, SeeAbility and Walk the Walk, to capture the essence of our annual summit in 2017 and what it is about our city of Cambridge which offers great opportunities to drive change in rare diseases.

The theme of CRDN2017 was ‘Re-imagining the Patient Journey’ and we worked hard to give patients and patient groups a platform and voice at the event through the Patient Journey Poster Exhibition and the Lightning Pitches element of the day.

For those who attended, take a look to see if you made it on film! For those who were unable to attend but are curious, watch the film and be inspired to attend this year’s RAREfest event – a weekend of celebration of everything rare on 30 Nov and 1 Dec 2018.

 

Watch the CRDN Summit Film 2017…

© Cambridge Rare Disease Network 2018
Website designed and built by Suzanne Morris using WordPress
Reg'd Charity Number: 1166365
Company Number: 9798317
Reg'd Office: 19 Sedley Taylor Rd, Cambridge, CB2 8PW
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Findacure reflections on CRDN2017 summit. Re-imagining the Patient Journey.

Findacure reflections on CRDN2017 summit. Re-imagining the Patient Journey.

On 23rd October the rare disease community gathered in Cambridge for the Cambridge Rare Disease Network’s third annual summit. This day-long conference is always a great opportunity for Findacure to meet up with the rare disease community on our own doorstep, and we were thrilled to be invited to speak on the day. Rather than give a blow by blow account of the day, Findacure’s CEO, Rick decided that his blog for the week would highlight some of its major themes.

All conferences are different. They have a different focus, target a different audience, and deliver a very different atmosphere. In a month filled with conferences, including Rick speaking at Bio2Business, an orphan drugs and rare diseases conference in London, and Katie attending EyeforPharma, it is the conferences focusing on rare diseases that have been particularly special. In addition to our own Newcastle Rare Disease Showcase event (more about that soon), the Findacure team had the pleasure of attending the CRDN summit.  After a great day, I thought that I’d share my thoughts on what the CRDN summit stands for.

 

C is for collaboration

Collaboration really was at the heart of the CRDN summit. The team had worked hard to select a programme of talks that showcased projects in the rare diseases that involved many different disciplines. The specialist orphan drug pharmaceutical company Sobi spoke about their work with patient groups, both to help develop new treatments, and to improve the way that drugs are delivered to ease their day-to-day lives.

The Mendelian team highlighted the work there online platform is doing to provide an effective digital diagnostic tool which is accessible and helpful to primary care professionals. Importantly this tool is responsive and relies on the input of clinicians around the world to improve its results.

Finally Kay Parkinson, CEO of Cambridge Rare Disease Network, gave an excellent talk about her personal experience as a mother of two children with Alström syndrome. When exhibiting a poster about the condition at an international conference, she made contact with a pharmaceutical company who had a product that had the potential to act as a treatment for this condition. From this serendipitous meeting, a research project and clinical trial for this ultra-rare condition has snowballed.

R  is for “Read all about it”

A great innovation of the conference, and real highlight for me, was the patient journey poster competition (a project born from Kay’s own poster experience). Prior to the conference, 33 different patient groups took the time to produce posters to summarise their rare condition and their own patient journey. These posters were shared with all delegates in printed form so that everyone, whether clinician, researcher, life sciences worker, or rare disease patient, could read all about the impact of a multitude of rare diseases.

Even more excitingly five poster authors were selected to give a five-minute lightning talk during the conference, telling the audience a little about their condition and their three wishes for the future. The CRDN team asked Findacure to chair this session, and Mary Rose and I brought our dreaded bell along to help ensure that all of the speakers kept to time. It was a real pleasure to be involved in this session, and the talks were of an exceptional quality. 

D is for diversity

The whole day was hugely diverse, with a number of different sessions helping to bring different interest groups into the rare disease fold. The team

from Students4RareDiseases were involved in a session with medics, students, and genetic counsellors focussing on the role early career healthcare professionals can play in rare diseases. There was also a session focussing on the role of technology in rare diseases, with the team from No Isolation showcasing a robot avatar, which is designed to help children who are unable to attend their school due to ill health remain engaged in the classroom. These two breakout sessions combined with the CRDN Companies Forum and patient journeys posters, really ensured that everyone had a place at the event.

N is for new hope

The whole event was a great showcase for rare diseases, and we are grateful to the CRDN team for giving Findacure such an exciting role in the day. Between the talks on drug repurposing, gene editing, and the impatient patient revolution, I left the day with the sense that there is lots to look forward to for rare disease patients in the near future.

© Cambridge Rare Disease Network 2018
Website designed and built by Suzanne Morris using WordPress
Reg'd Charity Number: 1166365
Company Number: 9798317
Reg'd Office: 19 Sedley Taylor Rd, Cambridge, CB2 8PW
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Join the CRDN on Jeans for Genes Day 22 Sept 2017

Join the CRDN on Jeans for Genes Day 22 Sept 2017

Join the Cambridge Rare Disease Network on Jeans for Genes Day!

Apply for your free fundraising pack to support Cambridge Rare Disease Network and Genetic Disorders UK.

Did you know that half a million children in the UK are affected by a life-altering genetic disorder? Jeans for Genes Day is an annual fundraising campaign, organised by Genetic Disorders UK (GDUK), to raise awareness and money to transform their lives. This year’s event will take place on Friday 22nd September, and to aachieve an even greater impact, GDUK are partnering with smaller genetic disorder charities like CRDN to help them spread the word, and in return, split the proceeds.

Would you or your school or workplace like to take part in this year’s Jeans for Genes Day for CRDN?

All you have to do is sign up on the official GDUK page, choosing Cambridge Rare Disease Network as your partner from the drop-down list, and encourage your pupils or colleagues to wear jeans on the day in exchange for a small personal donation. Extra fundraising iideas, such as cake sales or quizzes, are most welcome too!

Once you transfer the proceeds to GDUK, they will then transfer half to us for everyone we help to sign up. One exception – if you’ve taken part in Jeans for Genes Day in the last three years please DO  take part, but all of the money raised will go to GDUK.

As hundreds of schools and workplaces take part each year, this is a great way for your pupils and colleagues to feel like an important part of the wider movement to end genetic disorders. For schools, it is also an opportunity for you to educate your students about the science of genetics, to promote equality of people affected by rare conditions, and to highlight the importance of charity and social responsibility.

Need some help? The Jeans for Genes website is packed full of helpful resources like posters, sponsorship forms, educational leaflets, assembly ideas etc. for schoolsnurseries and workplaces a social media pack and arts and crafts ideas.

The of the money donated to us on Jeans for Genes Day will be used to help CRDN develop a regional network and forum of Rare Disease patients and their advocates and families, to support our Unique Feet children and young people’s social group and to run exceptional Rare Disease educational and networking events. Raising the profile of Rare conditions and valuing, celebrating and supporting those living with Rare Disease through life’s journey could be something you and your team or school could help us make this happen. If you have any questions about CRDN or Jeans for Genes Day, please don’t hesitate to get in touch.

 

 

© Cambridge Rare Disease Network 2018
Website designed and built by Suzanne Morris using WordPress
Reg'd Charity Number: 1166365
Company Number: 9798317
Reg'd Office: 19 Sedley Taylor Rd, Cambridge, CB2 8PW
To contact us please send an email
Our privacy & cookie policy
Through to the Finals!

Through to the Finals!

Havas Life Medicom a ‘specialist led, unified communications agency delivering connected healthcare communications, informed through fresh insights, smart thinking and inspired ideas’ have been supporting CRDN pro-bono over the last year in creating communications materials for our annual summit. They have done a superb job of listening to our needs, working with our small team and the odd hours they keep due to other work commitments, and in designing high quality promotional and delegate materials.

Havas Life Medicom entered CRDN’s Summit 2016 into the Communiqué Award’s 2017 and we’ve had the exciting news that it has been nominated as a finalist! This is a great privilege as these awards, and in particular, the ‘Excellence in Communications via a Live Event or Stand-Alone Activity’ category which the 2016 Summit was entered in, are always extremely competitive, so we will be up against some tough competition. Being shortlisted is a real achievement and we are all be very proud. The awards ceremony in on 6th July 2017, so we will keep you posted through social media.

© Cambridge Rare Disease Network 2018
Website designed and built by Suzanne Morris using WordPress
Reg'd Charity Number: 1166365
Company Number: 9798317
Reg'd Office: 19 Sedley Taylor Rd, Cambridge, CB2 8PW
To contact us please send an email
Our privacy & cookie policy
Parachute Jumping for CRDN

Parachute Jumping for CRDN

Six incredible ladies, Helena Baker, Zoe Napier, Emily Mortimer, Lisa Griffin, Caroline Churn and Juliette Newton will be parachute jumping for CRDN on the 4th June!
We’ll be there to cheer them on and watch them fly at Beccles Airfield, Norfolk. Please help our courageous ladies achieve their £1,800 fundraising target by donating through
 Helena’s Just Giving page, Emily’s Just Giving Page and Zoe’s Just Just Giving Page.

So why CRDN?

Helena Baker is Vice President of the Nursing Medical Research Network and was one of the key speakers at our event in March 2016, where we discussed the potential need for a rare disease nursing service. Helena was born with Fibular Hemimelia, a rare congenital disorder that left her, in her own words, with one leg shorter than the other– a tiny club foot, lots of missing ligaments in her leg and body, and a fierce determination not to let any of these problems stop her from doing what she wanted in life, except perhaps play hockey.

Helena recalls growing up having more operations than hot dinners, but never received a diagnosis. 

Eventually, just before she turned 50, she was told of her diagnosis. Doctors were clear there was nothing her mother could have done during pregnancy that would have changed the outcome. She is sure that having had a diagnosis earlier would have made a world of difference to her parents. Helena has been recovering from more surgery having had her right leg amputated below the knee last September and is currently learning to walk again. She is continuing to make a great recovery as she’s getting to grips with her new prosthetic leg.  Unfortunately there’s been a slight set back with Helena breaking her remaining ankle.

Helena says “On the day, I’m not allowed to jump wearing my prosthetic leg and I’m also not allowed to land on my recently broken one remaining ankle.  So I’m going to land on my instructor who will be delighted.  I’m planning on testing them out beforehand for looks and comfort.” Helena’s grit and good humour never cease to astound us. She reassured us  “We are approaching our impending doom in the spirit of total denial, all desperately trying to pretend that it’s not happening”.

That’s the spirit Helena!

Because of her strong belief that having access to a rare disease nursing service would have been a huge support to her and her family, she has decided to take on a parachute jump on Sunday 4th June 2017 at Beccles Airfield alongside Zoe Napier, Emily Mortimer, Lisa Griffin, Caroline Churn and Juliette Newton, to help raise funds for CRDN towards their goal of establishing such a nursing service for those with rare diseases.

Thank you for your support and good luck and a huge thank you to Helena & her team!

© Cambridge Rare Disease Network 2018
Website designed and built by Suzanne Morris using WordPress
Reg'd Charity Number: 1166365
Company Number: 9798317
Reg'd Office: 19 Sedley Taylor Rd, Cambridge, CB2 8PW
To contact us please send an email
Our privacy & cookie policy