About SOBI

Sobi (Swedish Orphan Biovitrum Ltd) is a leading integrated biopharmaceutical company dedicated to bringing innovative therapies and services to improve the lives of rare disease patients and their families.

Learn more: www.sobi.com

“For the hundreds of children, young people and families in our care, with the most rare and complex conditions imaginable, life can be difficult - seeing multiple specialist teams at different times and in different places, impacting on day to day life and causing additional stress and anxiety.

“Joining our experts together in our fabulous new Rare Diseases Centre will allow us to bring all of these appointments together - vastly improving diagnosis, treatment and research, and ensuring all our children and young people get the very best care available anywhere in the world."

Sarah-Jane Marsh, Chief Executive at Birmingham Women’s and Children’s NHS Foundation Trust


 

"We are delighted that Birmingham Children’s Hospital are creating the first ever Children’s Rare Disease Centre. As the national charity supporting families affected by tuberous sclerosis complex, we see the difficulties patients can face in accessing expert, coordinated care.  The Children’s Rare Disease Centre will make such an enormous difference to so many young lives. We are so very grateful to Larissa for the support she has given to our community and for all that she does for children with rare diseases and their families."

Jayne Spink, Chief Executive TSA


 

"Having had two children with a rare disease who were not diagnosed for 18 years- the need for a specialist center is very clear to me. Dr Larissa Kerecuk is the perfect physician to lead this project, she is kind, understanding and above all a great champion for children's with rare diseases needs."

Kay Parkinson (LLB Hons), Chief Executive Officer, Cambridge Rare Disease Network (CRDN)


 

"As Chair of the Houses of Parliament All-Party Parliamentary Group on Rare, Genetic and Undiagnosed Conditions, I am absolutely delighted with Birmingham Children’s Hospital vision of a dedicated centre for children and their families with rare conditions. Dr Kerecuk has done amazing work to turn this vision into a reality.

Birmingham Children’s Hospital new rare disease centre will provide support to families who have rare and undiagnosed conditions and compliment the wonderful work undertaken everyday by staff in the NHS. 

By stimulating much needed research into the mechanisms of disease and the development of new treatments for these conditions, this new centre will encourage collaboration across the NHS and Universities so helping cement the UK’s position as a world leader in innovation and healthcare provision."

Ben Howlett, Chair All Party Parliamentary Group (APPG) on rare, genetic and undiagnosed conditions


“Having this children’s Rare Diseases Centre represents a world’s first for Birmingham. Apart from enabling us to provide a multidisciplinary service offering high quality holistic care to these patients and their families, it will also substantially reduce the amount of time spent attending hospital, including multiple outpatient appointments.

"The appointment of the inspirational Dr Larissa Kerecuk as both Rare Diseases Lead and also Regional Paediatrics research Lead offers an unique opportunity to combine both really high quality clinical care with cutting-edge research”

Professor Jeremy Kirk, Clinical Director at the National Institute for Health Research Clinical Research Network West Midlands, and Director of Research and Innovation, Consultant Paediatric Endocrinologist, Birmingham Children’s Hospital


 

"Having the Children’s Rare Disease Centre here in Birmingham is such a fantastic opportunity for providing and researching better treatments for patients in the West Midlands. With Dr Larissa Kerecuk as the Rare Disease Lead and also the Children’s Research Specialty Lead for the NIHR Clinical Research Network (CRN), we have a real synergy that will enable greater research within the NHS for children with rare diseases. A unique and exciting initiative that the National Institute for Health Research (NIHR) is pleased to support. "  

Dr. Linda Porter, Research Delivery Manager, CRN West Midlands


 

“The 100,000 Genomes Project would not be possible without the support and dedication of clinicians and healthcare professionals in the NHS. I would like to thank Larissa and the Birmingham Children’s Hospital team for their tremendous hard work. They have enabled more families affected by rare diseases to take part in this project, and our aim is offer diagnoses and better treatments for these families. The new Rare Disease Centre for children will build on Birmingham Children’s excellence in care and expert clinical knowledge to truly transform care for patients now, and in the future.”

Professor Mark Caulfield, Chief Scientist at Genomics England


 

"The Children’s Rare Disease Centre in Birmingham is the first to deliver individually tailored multidisciplinary clinical services for children with rare diseases. These will screen for complications, offer advice on medical management, and a holistic, whole person approach, to support the child, their carers, and their local medical teams to give them the best possible care. Equally importantly, it will link seamlessly with our children’s NIHR Wellcome Clinical Research Facility to support trials of new treatments; and with our adult rare disease centre, so that children do not get lost in the transition to adult care. Dr Larissa Kerecuk has done a fantastic job leading this initiative, and is the epitomy of a caring children’s doctor."

Timothy Barrett, Rare Disease Lead BHP


"Rare diseases are often complex multi-system disorders needing input from a wide range of different clinicians for them to be managed effectively. Too often the care that families affected by rare diseases receive from the NHS requires them to rattle round the system, seeing different doctors in different hospitals on different days. The creation of a specialist multi disciplinary centre for rare diseases under the inspirational leadership of Dr Larissa Kerecuk shows that this does not have to be the case. This multi million pound development will ensure that families can receive the multi-disciplinary care they need without having to traipse from pillar to post.It will also build expertise and promote research, so families in future will benefit from the new knowledge and better ways of working that have been made possible by the creation of this centre. Dr Kerecuk's brainchild is an example that the NHS should emulate elsewhere when planning services and support for the 3.5 million people affected by rare diseases across the UK today."

Alastair Kent OBE, Director, Genetic Alliance UK


 

"Dr Kerecuk and her colleagues at Birmingham Children's Hospital can be proud of developing the first Children's Rare Disease Centre and Service in the world in Birmingham which also has the Centre for Rare Diseases for adults. This will benefit patients and families affected by rare diseases in our fantastic city of Birmingham as well as those around the world by the new progress made."

Gisela Stuart MP Birmingham Edgbaston


 

"The soon to be opened  paediatric rare disease centre at Birmingham Children's Hospital is a much needed leap forward in the holistic approach to treating and supporting children and families affected by rare disease.

"Led by the inspirational Dr Larissa Kerecuk, the centre will be a model hopefully for further centres across the UK and beyond."

Richard Lynn, BPSU Scientific Coordinator, UCL Hon Senior Research Associate, British Paediatric Surveillance Unit


 

“The team at Birmingham Children’s Hospital recognise that the care and treatment of patients is more than the provision of medical care.  Being affected by a rare disease can be isolating and a scary world to inhabit.  They recognise this and are leading the way by ensuring there is a specialist centre that truly understands the needs of the whole family.  The Rare Disease Centre will ensure that from the moment you step through the door you will know that you are part of a team who truly understands you and your condition.  Same but Different are proud to be working with the hospital to offer people an insight into the rare world through our photography project Rare Beauty.”

Ceridwen Hughes ‘Same but Different’ project


"That patients and their families lie at the heart of the research and innovation led Birmingham Health Partners is a reflection of the work that our shared community does to ensure that the best care and the latest insights gained through research are accelerated through to their benefit.

"I am therefore delighted to be able to join the community in offering my congratulations to Larissa Kerecuk and the wider BCH team who have made the establishment of this centre possible."

John Williams, Managing Director - Birmingham Health Partners


 

"Roald Dahl’s Marvellous Children’s Charity is delighted and privileged to be working with Dr Larissa Kerecuk who continues to work tirelessly in the field of treatment, care and support for young people and their families with rare and sometimes undiagnosed illnesses. 

"Our three paediatric rare disease clinical nurse specialists at Birmingham Children’s Hospital are helping to lead the way in designing a service which is at the forefront of care for children with these conditions.  We believe our partnership is a fantastic example of how the NHS and the third sector can work hand in hand, focusing on the same goals and together, achieving the results that our young people and their families deserve."

Jane Miles, CEO, Roald Dahl’s Marvellous Children’s Charity


 

"We are pleased that the centre will be in Birmingham as this will add to the outstanding expertise already in place. We are sure this will provide new opportunities for people within the rare disease community and promote the ever-growing need for ongoing research and development.

"Our thanks go to Larissa and her team who have worked tirelessly to ensure that patients have had a voice in the development of the centre, we were involved in the very early consultations about the design of the building to put patients and their families at the very heart of this new Centre."

Ann Chivers CEO of Alström Syndrome UK


 

"Under the expert guidance of Rare Disease Lead Dr Larissa Kerecuk, Birmingham Children’s Hospital is the top paediatric recruiter for RaDaR - the National Registry of Rare Kidney Diseases (). RaDaR was co-founded in 2010 by Dr Kerecuk’s predecessor Dr Mark Taylor and Prof Moin Saleem from Bristol Children’s Hospital. Over the past 7 years it has developed into a leading resource for researchers investigating rare kidney diseases, with more than 10,000 UK patients recruited to date, covering over 50 conditions. Plans are now in place to expand RaDaR to cover non-renal rare conditions. Birmingham Children’s Hospital will be the lead site for this expansion.

"Dr Kerecuk is a tireless advocate of both rare disease research and family support, organising a series of very well attended family information days. The new rare disease centre, the first in the UK, will revolutionise both the care and personal experience of rare disease patients and their families, bringing a holistic, patient-centred approach to this much needed area of care."

Melanie Dillon, Rare Disease Operations Manager, UK Renal Registry


"The paediatric rare disease centre at Birmingham Children’s Hospital offers a unique opportunity to provide the care and support that children diagnosed with some of the world’s rarest conditions desperately need.

"The opportunity to house experts in all medical specialities in one centre, combined with the facilities to make a hospital visit a less stressful experience, will transform treatment for these patients. This centre can help drive research into rare diseases, improve our understanding of conditions, and help to build patient communities. We hope it will provide a template for rare disease treatment in the future.

"Dr Larissa Kerecuk is an exceptional lead for the rare disease centre. She is dedicated, passionate and enthusiastic, and understands that the opinions and experiences of patients are just as important as those of clinicians."

Findacure

 

Sobi (Swedish Orphan Biovitrum Ltd)

We care about rare disease patients.
Sobi is a leading integrated biopharmaceutical company dedicated to bringing innovative therapies and services to improve the lives of rare disease patients and their families.

www.sobi.com