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Research study exploring views and attitudes to stem cell therapy for Osteogenesis Imperfecta

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A new research study called Boost Brittle Bones Before Birth (BOOSTB4) is investigating the possibility of using stem cell therapy as treatment for severe OI. The study is being undertaken by researchers at Great Ormond Street Hospital and the UCL Institute of Child Health. 

The treatment involves transplanting stem cells into the baby affected with OI during pregnancy or soon after birth. The stem cells will make healthy collagen that will support damaged bones and help with the development of new normal bone. The BOOSTB4 clinical trial aims to test whether the treatment is safe and effective.

Great Ormond Street Hospital & UCL Institute of Child Health feel it is important to find out what people think of this new treatment and would like to invite people to give their views in an interview (phone or face-to-face) or focus group.

 

They would like to hear from;

  • Young people (ages 16 -18) affected with OI
  • Adults affected with OI
  • Parents and carers of children affected with OI
  • Health professionals and patient advocates who work with people affected with OI

 

If you are interested in taking part in an interview and would like to find out more about the study, please contact Dr Melissa Hill at Great Ormond Street Hospital by email melissa.hill@ucl.ac.uk or phone 020 7762 6871.    


 

TOPaZ Trial

If you have a diagnosis of Osteogenesis Imperfecta and are aged 18 years or over, you may be eligible for the Treatment of Osteogenesis Imperfecta with Parathyroid hormone and Zoledronic Acid (TOPaZ) Trial across the UK and Ireland. The study aims to determine whether a two year spell with a treatment called Teriparatide (TPTD) followed by treatment with another drug called Zoledronic Acid (ZA) reduces the risk of broken bones occurring in people with Osteogenesis Imperfecta (brittle bone disease) as compared to standard care.

If you would like to find out more information, please contact the research team:

TOPaZ Trial Office - Holly Ennis

0131 537 2573 or topaz.trial@ed.ac.uk

Alternatively, please take a look at the website: edin.ac/topaz-trial or download the flyer

 

 

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TOPaZ is a non-commercial trial sponsored by a partnership of the University of Edinburgh and NHS Lothian called ACCORD and funded by the UK National Institute for Health Research (NIHR). 


 

 

James Lind Alliance (JLA) Priority Setting Partnership 

Help set the research agenda for rare musculoskeletal diseases in adulthood


Created to identify unanswered questions about Osteogenesis Imperfecta and other rare conditions which affect the bones in adults, the JLA is bringing together patients, carers and social care professionals in Priority Setting Partnerships (PSPs) to agree what research matters most to patients and health care professionals.

What questions do you think need answering in the area of Osteogenesis Imperfecta?

Have your say; participate in the survey and help set the priorities for research in OI: https://www.surveymonkey.co.uk/r/JZ9RDFK

 

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LINK Clinical Trial 

Brittle Bone Society would like to make you aware of The LINK Study which aims to find out what impact the investigational medication has on the participant’s bone mineral density and the frequency of fractures in children and adolescents with OSTEOGENESIS IMPERFECTA. If you would like more information see contact info below.

 

 

http://brittlebone.org/files/6014/8758/9651/023_AMG_20130173_FlyerBBS.pdf

 

RUDY Study

RUDY - The Rare UK bone, joint and blood vessel disease study - will develop detailed phenotypes of patients with rare musculoskeletal conditions to identify subgroups of patients with these conditions, and provide a platform from which collaborations with the NHS, industry and academia can be established to develop new tests and treatments.

For the study a secure web interface based database will be used to collect and store data, and as the primary mode of interaction with study participants.

"Being involved in this new study, where we have a few basic medical tests and fill in questionnaires on a database, is an exciting opportunity for all of us. Our involvement, as individuals, will potentially work towards getting better treatment for OI in the future - and how fantastic would that be?! I hope as many members as possible will take this opportunity up, as the more information provided the better the study will be." - Elaine Rush

Find RUDY Study at Web / Facebook / Twitter


 

Scoliosis Research                   

SCOLIOSIS RESEARCH PRIORITY SETTING PARTNERSHIP - has been set up to identify unanswered questions about the diagnosis and management of scoliosis. The James Lind Alliance have been in touch with the BBS to ask if our membership would like to help them in an important study. The survey is available at https://www.surveymonkey.co.uk/r/SSJZ28R or contact the James Lind Alliance Project Manager at the Oxford Biomedical Research Centre to request a paper version (voicemail 01865 223298, e: sandra.regan@ouh.nhs.uk

You can find more information here

UK Clinical Trial Gateway 

By clicking on this link you can find out what clinical trials are happening across the UK. Every year, more than half a million people help the NHS improve healthcare and develop life-saving treatments by taking part in clinical trials. Whether you are a patient or a healthy member of the public, there are many reasons why you might decide to take part. click here

 


 

The 100,000 Genomes Project

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A UK government project is now running under the NHS called the 100,000 Genomes Project to find the genes that cause rare diseases such as Osteogenesis Imperfecta.

 

Who is eligible?

Currently only patients living in England with Osteogenesis Imperfecta. This particularly applies if no one in your family has had genetic testing or had a gene test but no cause was found. Only one person from each family with OI needs to join.

 

How do I join? 

See your GP or hospital team and ask to be referred to your nearest genetics clinic running the project.

 

What happens when I am seen at the genetics clinic?

You will be given more information about the project and an opportunity to ask any questions. If you choose to take part you will fill in some consent forms and be asked to give a blood sample.

The results may take some time to be available but will be sent to you and your doctor.

 

What is the benefit of taking part?

Results may take some time but will be sent to you and your doctor and will find out the underlying cause of your OI. Results will then be used to improve and help our understanding of Osteogenesis Imperfecta for future generations.

 

How do I find out more?

Check out the website www.genomicsengland.co.uk and take a look at the information sheet. 




 

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We provide information on our website for general information only. The content is not intended to be and should not be used as a substitute for professional medical advice, diagnosis or treatment. Users should not take any action or refrain from taking any action because of any information on our Website. Users should always seek the advice of their doctors and of other qualified health professionals concerning OI and other medical conditions. So far as permissible by law, the Brittle Bone Society does not accept any liability to any person relating to the use of any information on this Website.