Personalize My Medicine is partnering with Students 4 Rare Diseases!

Personalize My Medicine is proud to be partnering with Students 4 Rare Diseases (S4RD)!

students4rarediseases is a committee that connects rare disease societies from different medical schools in order to share speaker information and advertise events. We originally founded Barts and The London Society for Rare Diseases in 2011 and expanded the project in the form of S4RD in 2013 so that we can help students in other medical schools to get involved. Our main aim is to create a network in order to pool resources and information about rare diseases. If you would like to get involved please contact us. You can attend events, support the rare disease society at your own medical school or even start your own society if there isn’t one.

Well done to CortiCare for achieving 3rd place in Innovation Forum´s IMAGINE IF! Competition!

With Dr Steven Hilton and Marpin Kho, co-founders of ART Research, at Innovation Forum´s IMAGINE IF! competition

It has been a great experience mentoring CortiCare and Art Research for Innovation Forum´s IMAGINE IF! competition! If you are a health or medical innovation start-up, get in touch with PMM to find out about participating in our Start-up Showcase! Like our new PMM Start-up Showcase page on Facebook to keep up to date on the latest health start-ups. https://www.facebook.com/PMM-Start-up-Showcase-454294308286852/

 

Delivering Outstanding Rare Care: Q&A Released with Rare Disease revolutioner, Larissa Kerecuk, Ahead of Orphan Drugs Conference


Orphan Drugs & Rare Disease UK, 18th-19th October, 2017, London UK

Rare disease expert speaks to SMI Group in the run-up to her featured address on the 100K Genome

SMi Group are thrilled to release an interview with Larissa Kerecuk, ahead of her presentation on Developing the 1st Rare Disease Centre for Children in the World at the 7th annual conference on Orphan Drugs and Rare Diseases this Autumn. With a wealth of experience and devotion to leading the development of the first pediatric Rare Disease Centre in the World, providing a diagnosis and personalised approach to many yet undiagnosed children, Larissa is a rare disease renowned revolutioner known for promoting a holistic approach.

As the founder and coordinator of the Autosomal Recessive Polycystic Kidney Disease Rare Disease Group in the UK, Larissa and her team have established international collaborations with the US and Europe, which has also developed the Human Factors Course to improve patient safety. With patient safety as her driving force, Larissa became NIHR Clinical Specialty Lead for Paediatrics in the West Midlands to work towards her vision of research become an integral part of everyday NHS care. Focusing on collaboration, the presentation will offer attendees with guidance into the importance of research and collaboration for progression in the rare disease field.

Understanding the major challenges of rare diseases faced by people affected by rare disease will be a key focus, as will hearing about patient history – a patient speaks and the development of the 100K Genome, will be just some of the highlights in her talk.

“The perspective that I am bringing to the conference is from the clinicians and patients within the NHS with all the challenges that families affected by rare diseases must deal with on a daily basis: from getting a diagnosis to being able to have some form of treatment for the patient and the effect the rare disease has on the whole family”

In the run up to the event, SMi Group caught up with Larissa to discuss her view of the orphan drug landscape and how she sees it evolving in the upcoming years.

“…This is both an exciting and challenging time for the orphan drugs industry: there are so many different possible treatment avenues becoming apparent with the genomic era of knowledge but challenging in terms of fast-changing global regulations and markets. There is huge growth potential as the new genomic knowledge is explored and built upon. Improvement is required from all stakeholders to work more collaboratively to achieve therapeutic benefits to patients as quickly and safely as possible…”

The full interview is available to read in the event download centre.

Running alongside the conference will an exclusive pre-conference workshop held on Tuesday 17th October 2017.

WORKSHOP: Rare Diseases Challenges and Opportunities

Workshop Leaders: Larissa Kerecuk, Rare Disease Lead, Consultant Paediatric Nephrologist, Birmingham Children’s Hospital & Janet Tuberville-Greenlay, Roald Dahl; Rare Disease Transitition Sister, Birmingham Children’s Hospital

For sponsorship packages: Contact Alia Malick +44 (0) 207 827 6168 or e-mail amalick@smi-online.co.uk.

For delegate enquiries: Contact Sean Vaghela on +44 (0) 20 7827 6744 or email saghela@smi-online.co.uk For media enquiries, contact Kyra Williams on +44 (0) 20 7827 6012 or email kwilliams@smi-online.co.uk

London, UK www.orphandrugs.co.uk/PMM Contact e-mail: kwilliams@smi-online.co.uk Contact tel: +44 (0) 207 827 6012 #smiorphandrugs

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About SMi Group: Established since 1993, the SMi Group is a global event-production company that specializes in Business-to-Business Conferences, Workshops, Masterclasses and online Communities. We create and deliver events in the Defence, Security, Energy, Utilities, Finance and Pharmaceutical industries. We pride ourselves on having access to the world’s most forward thinking opinion leaders and visionaries, allowing us to bring our communities together to Learn, Engage, Share and Network. More information can be found at http://www.smi-online.co.uk

2017 on Track to be a Record Breaking Year for Orphan Drug Approvals

 

Orphan Drugs & Rare Diseases UK, 18th-19th October, 2017, London UK

“…The U.S. FDA is on track to set a record for orphan drugs approved this year. At last count, 42 orphan drugs have been approved between January 1, 2017 and August 9, 2017. At their current pace, the FDA will approve more than 60 orphan drugs by the end of the year. That is 20% above their last record breaking period in 2014 (49 approvals) and 2015 (48 approvals) …”*

2017 is on track to be a record-breaking year for Orphan Drug approvals. The 7th annual event is a true testament to how the industry has grown. Now, in 2017, this year’s Orphan Drugs and Rare Diseases conference will be a fusion of exciting new content and dynamic discussions featuring 20 industry-led and case-study driven presentations and hands on training including an inspiring workshop with the ability for 1-2-1 learning. This year is set to be the biggest and best Orphan Drugs event yet! Join the conversation about key strategies to accelerate rare disease clinical drug development; expand your knowledge on patient collaboration and patient centric models; address challenges that come with working on rare diseases, as well as the development of the world’s first rare disease centre for children; and to hear regulatory challenges for orphan products.

Attending Orphan Drugs and Rare Diseases UK 2017 will give you the chance to converse with CEOs, Chiefs, VPs, Directors, Heads, and Principal Scientists, dedicated to working on the continued improvement of orphan drug development and rare disease research.

Confirmed attendees include: Action Duchenne, ADB Medical, Alexion Pharma GmbH, Ataxia UK, Birmingham Children’s Hospital, Cambridge Rare Disease Network (CRDN), Chiesi Farmaceutici S.p.A., Deutsches Krebsforschungszentrum, Findacure, Healx, High Force Research, Idorsia Pharmaceuticals Ltd, MEDExpansion, MPS Society, National Institute for Health and Care Excellence, Philip Chapper, Sanofi, Santhera Pharmaceuticals, Selecta Biosciences, Summit Therapeutics, The Medical Research Network, University Of Groningen, Vertex Pharmaceuticals, and many more!

Running alongside the conference, will an exclusive pre-conference workshop held on Tuesday, 17th October 2017.

WORKSHOP: Rare Diseases Challenges and Opportunities

Workshop Leaders: Larissa Kerecuk, Rare Disease Lead, Consultant Paediatric Nephrologist, Birmingham Children’s Hospital & Janet Tuberville-Greenlay, Roald Dahl; Rare Disease Transition Sister, Birmingham Children’s Hospital *SOURCE: http://bit.ly/2vEHi9O For sponsorship packages: Contact Alia Malick +44 (0) 207 827 6168 or e-mail amalick@smi-online.co.uk.

For delegate enquiries: Contact Sean Vaghela on +44 (0) 20 7827 6744 or email saghela@smi-online.co.uk For media enquiries, contact Kyra Williams on +44 (0) 20 7827 6012 or email kwilliams@smi-online.co.uk

London, UK www.orphandrugs.co.uk/PMM Contact e-mail: kwilliams@smi-online.co.uk Contact tel: +44 (0) 207 827 6012 #smiorphandrugs

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About SMi Group: Established since 1993, the SMi Group is a global event-production company that specializes in Business-to-Business Conferences, Workshops, Masterclasses and online Communities. We create and deliver events in the Defence, Security, Energy, Utilities, Finance and Pharmaceutical industries. We pride ourselves on having access to the world’s most forward thinking opinion leaders and visionaries, allowing us to bring our communities together to Learn, Engage, Share and Network. More information can be found at http://www.smi-online.co.uk

Personalize My Medicine is proud to announce a partnership with Raremark!

Personalize My Medicine is partnerning with Raremark!
Raremark keeps families updated with the latest research and treatments in development, and enables them to share their valuable experience to help others. We also help people affected by a rare disease be heard by the people developing new treatments. Our aim is to bring the voice of the community into the development of new medicines, so they are produced with families in mind. Find out more at www.raremark.com
 
New Tool!
In a recent survey conducted by Raremark with people affected by a rare disease, 94% of participants said they would share their data to help others. Raremark has built a new tool that helps families share their valuable real-world experience which can help to speed up drug development. To sign up and start sharing your experience in rare disease in on of our nine rare disease communities, click here. We are adding new communities every month.

 

Free Webinar on Qualification of Biomarkers!

Complimentary Webinar from the Predictive Safety Testing Consortium and Merck & Co, Inc

8th August 2017, 5 pm. BST

Presented by John-Michael Sauer, Executive Director, Predictive Safety Testing Consortium, Critical Path Institute & Xuemei Zhao, Senior Principal Scientist, Translational Molecular Biomarkers, Merck & Co., Inc.

Register today, for free: http://bit.ly/2sFP9Sg

Can’t make the dates? Still register and a copy of the recording will be sent to you

Biomarkers form a pivotal role in drug development today and as biomarkers become integrated into drug development and clinical trials, quality assurance and in particular assay validation becomes essential. There is a need for standardised guidelines for analytical methods used in biomarker measurements. Also, new biomarkers used for both the development and use of therapeutics are dependent on the establishment of a solid validation process that addresses technology integration and method validation as well as regulatory pathways for efficient biomarker development. The webinar focuses on the scientific and regulatory considerations for the analytical validation of assays in clinical trials. It will also address recent regulatory updates and the standardisation of validation procedures for efficient biomarker development.

Benefits to attending the webinar:

  • Advance your knowledge on Scientific and Regulatory Considerations for Biomarker validation
  • Boost your understanding of Analytical Validation of Assays Used in the Clinical Qualification of Biomarkers

Can’t make the dates? Still register and a copy of the recording will be sent to you.

For further information on how to save on your attendance and what discounts we have available please contact a.fernandez@oxfordglobal.co.uk

Personalize My Medicine is proud to be partnering with FindACure!

Personalize My Medicine is partnering with FindACure!

 

Findacure is a UK charity that builds the rare diseases community to drive research and develop treatments.
Rare diseases are defined in the EU as conditions affecting less than 1 in 2000 people — though they come much rarer than this. Because of their small patient populations, rare diseases are often assumed to be unimportant. This leads to a lack of support for patients and families, neglect by the medical profession, and little ongoing research into treatments. Findacure is here to help. We empower rare disease patient groups through a series of training programmes, and encourage collaboration between rare disease stakeholders to advance research. 

 

 

Personalize My Medicine is partnering with SMi´s 7th Annual Orphan Drugs & Rare Diseases conference!

Personalize My Medicine is please to announce a partnership with SMi´s 7th annual Orphan Drugs & Rare Disease conference!
7th annual Orphan Drugs and Rare Diseases UK
18th & 19th October 2017
Holiday Inn Kensington Forum, London, UK
Organisers: SMi Group
Tel: +44 (0) 207 827 6012
http://
www.orphandrugs.co.uk/PMMed

SMi Group is thrilled to present the 7th annual Orphan Drugs and Rare Diseases conference, taking place on 18th & 19th October 2017 in Central London, UK. This year’s theme will be focused towards discussing strategies for patient engagement, market access and gene therapies to enhance rare diseases and orphan drug research.
 
Aimed at an audience of senior scientists and oncology specialists involved in targeting rare diseases therapies and drug research, Orphan Drugs UK 2017 will provide a perfect platform to discuss pioneering clinical developments and the next generation of clinical trial process. The 7th annual conference will capture expert insight through dedicated focus new therapies for different rare diseases, the importance of orphan drug development and reimbursement and repurposing.
 
Join us this October for strategic direction from the Rare Disease community and leverage your knowledge with the key requirements and tools for successful patient recruitment and retention through informed guidance delivered by a panel of industry experts.
 
Featured Speakers:
  • Olivier Morand, Clinical Science Program Head, Actelion Pharmaceuticals
  • Larissa Kerecuk, Rare Disease Lead, Consultant Paediatric Nephrologist, Birmingham Children’s Hospital
  • Oliver Timmis, CEO, AKU Society
  • Stuart Hughes, Director, Head of Pharmacology, Vertex
  • Nadia Assenova, Senior Director Regulatory Affairs, EMEA, Alexion Pharma GmbH
  • Christine Lavery, Group Chief Executive, MPS Society
  • Hsin Loke, Director, Strategy, Operations and Finance, Rare Diseases Unit, GlaxoSmithKline
  • Olaf Ritzeler, External Innovation Lead, Sanofi
  • Stephen Marcus, CEO, Cantex Pharmaceuticals
  • Kei Kishimoto, Chief Scientific Officer, Selecta Biosciences
Exclusive highlights in 2017:
  • Learn about new therapies for different rare diseases, and how these can successfully be applied to other diseases with similar attributes
  • Hear what opportunities and challenges come with working on rare diseases, as well as the development of the world’s first rare disease centre for children
  • Expand your knowledge on patient collaboration and patient centric models with the stream on patient engagement, covering improvement strategies, patient-led trials, and much more
  • Gather further insight on drug approval and reimbursement with MAA, and how techniques for drug repurposing in the rare disease area can help treatment