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Date: 2017-10-21


Date: 25 NOVEMBER 2017 - 2PM TO 5PM

Venue: Siobhan Davies Dance Studio, 85 St. George’s Rd, London, SE1 6ER

RSVP by 20/11/17: contact@pnhuk.org

Refreshments provided. Family members/carers welcome.

Date: 2017-08-14

The Newcastle Rare Disease Showcase

Join Findacure for The Newcastle Rare Disease Showcase, a day-long event to celebrate and highlight rare disease projects taking place in Newcastle and the North East.

By providing a friendly forum for patient groups, researchers, clinicians and life science professionals to come together, we hope to encourage collaborations within the rare disease community to improve the future of rare disease science and healthcare.

The showcase will take place on Tuesday 31st October, 10:00 - 16:30, at the Centre for Life in Newcastle. The day will feature presentations on the latest developments taking place in the rare disease community, and include talks on rare bone disorders, neuromuscular disorders and rare skin cancer. The event will also include a two-hour networking session, where delegates will have the opportunity to propose their own five-minute lightning talks.



Date: 2017-08-14

Webinars from AAMDSIF

Here are links to previous webinars delivered by AAMDSIF (USA based Foundation supporting those with AA, MDS and PNH)

The PNH Patient’s Journey: A Snapshot of Short- and Long-Term Effects https://www.pathlms.com/…/co…/4958/video_presentations/71565

Understanding Personalized Medicine for Aplastic Anemia, MDS and PNH Patients https://www.pathlms.com/…/co…/5041/video_presentations/74444


Date: 2017-06-04

AAMDSIF Webinars

June 29th 2017 - Understanding Personalized Medicine for Aplastic Anemia, MDS and PNH Patients

Thursday, June 29th 
1:00 – 2:30 PM EST (note this is USA time)
REGISTER here: https://www.pathlms.com/aamdsif/webinars/2236


Dr. Patel from MD Anderson Cancer Center will explain the basics of diagnostic genetic testing and its significance in bone marrow failure diseases. Dr. Patel will also discuss how these tests will impact future treatments and therapies for aplastic anemia, MDS, PNH and other related bone marrow failure diseases such as AML, CMML, MPNs, and PRCA. This webinar is brought to you by Celgene, Achillion, Takeda and Genentech.

14th June 2017 - The PNH Patient’s Journey: A Snapshot of Short- and Long-Term Effects

Wednesday, June 14
1:00 – 2:30 PM EST (note this is USA time)
REGISTER here: https://www.pathlms.com/aamdsif/webinars/2058

Registered nurse Pierse Byrnes from Johns Hopkins University will discuss the disease’s short- and long-term effects on PNH patients. This will include a brief explanation of the causes of PNH, along with common side effects you may experience and ways to manage your symptoms for a better quality of life. Pierse will conclude with frequently asked questions that you can pose to your doctor and healthcare team. This webinar is brought to you Achillion Pharmaceuticals and Genentech.


Date: 2017-05-06

Clinical Trials Day 2017 - Kings College Hospital London Programme

Thursday 18 May 2017

Session on PNH/AA Clinical Trials between 10am and 11am in the Large Meeting Room, Ground Floor of the James Black Centre (Denmark Hill campus). 


Date: 2017-05-03

PNH Support Annual General Meeting (AGM) - Saturday 10 June 2017 - Save the Date

PNH Support - AGM - Saturday 10 June 2017 - 11am followed by lunch and an opportunity to meet other patients/family members (this may have a formal structure if people would like -tbc).

Venue: National Council for Voluntary Organisations ( NCVO) Society Building, 8 All Saints Street, London, N1 9RL (same venue as the Patient and Family Conference last year).

To attend the AGM and vote on resolutions, you must be an "official" member of PNH Support. PNH patients, their family members and carers are eligible to become members. Membership is free and members receive a 6 monthly newsletter and email updates on recent developments. Members can be involved in the charity as much or as little as they like. Members are required to provide us with their address for our Register of Members (as required by the Charities Commission). The more "official" members we have, the stronger our collective voice is when advocating on behalf of our members so we would encourage as many of you as possible to sign up!  Being a member of the closed Facebook page is not the same as being a member of the charity. If you would like to become an official member of PNH Support, please email us at contact@pnhuk.org with your details and postal address.

Even if you don't attend the AGM, PNH patients and family members are welcome to attend the lunch and gathering afterwards. More details will follow.


Date: 2017-03-20

The Cardiff Rare Disease Showcase

Findacure is pleased to announce The Cardiff Rare Disease Showcase!  
Taking place on Thursday 4th May at the Life Sciences Hub Wales, our networking event will bring together key stakeholders from the rare disease community to share ideas, encourage research and highlight rare disease projects happening in Wales. 

The evening will include case studies from the ED Society and Tuberous Sclerosis researchers, alongside a selection of five minute lightning talks proposed by delegates. The event will be supplemented by canapés and refreshments to create an informal atmosphere and promote open conversation.

Tickets are on sale now at Eventbrite - https://www.eventbrite.co.uk/e/the-cardiff-rare-disease-showcase-tickets-32688281530

Registration is free for patients, patient groups and charities.


Date: 2017-01-14

Events in the UK on Rare Disease Day 2017 - 28 February 2017

If you are interested in attending events to celebrate Rare Disease Day on 28 February 2017 please see here to register:


Date: 2017-01-02



Meetings MONDAYS, every 6-8 weeks, for coffee/tea and supportive sharing of PNH concerns. We will occasionally join the MDS group for talks by professionals on related blood conditions.


2017 Meetings

Dates: 23 January, 20 March, 22 May, 17 July, 18 September, 20 November

Time: 10.30 – 12.30

Venue: Cambridge Cancer Help Centre, David Rayner Building, Scotsdales Garden Centre,

120 Shelford Road, Cambridge CB22 5JT (free parking)


Please contact Caroline Fookes (01763 838045 or carfoo@waitrose.com),

or Maria Piggin (contact@pnhuk.org), for further information

Date: 2016-09-10

London Patient Meeting - 5 November 2016

We are holding a patient/family meeting in London on Saturday 5 November between 10am and 1pm at the Siobhan Davies Studios near Elephant and Castle (the same venue where Kings College Hospital used to organise meetings). The address is 85 St. George’s Road, Elephant & Castle, London, SE1 6ER. I know this won't be convenient for everyone - if you want to volunteer to find an appropriate local meeting venue in your region and offer to initiate the meeting (with our help), please do let us know. Please RSVP by 15 October 2016 to contact@pnhuk.org.

Date: 2016-09-10

Anglia Region PNH Patient and Family Meetings  

MONDAYS on a 6-8 week basis for coffee/tea and a chat and to discuss ideas for future meetings and support

3rd October 2016, 5th December 2016

10.30am – 12.45pm

Venue: Cambridge Cancer Help Centre, David Rayner Building, Scotsdales Garden Centre, 120 Shelford Road, Cambridge CB22 5JT (free parking). Please contact Caroline Fookes (carfoo@waitrose.com), or Maria Piggin (contact@pnhuk.org), for further information.

MONDAYS on a 6-8 week basis for coffee/tea and a chat and to discuss ideas for future meetings and support

3rd October 2016, 5th December 2016

10.30am – 12.45pm

Venue: Cambridge Cancer Help Centre, David Rayner Building, Scotsdales Garden Centre, 120 Shelford Road, Cambridge CB22 5JT (free parking). Please contact Caroline Fookes (carfoo@waitrose.com), or Maria Piggin (contact@pnhuk.org), for further information.

Date: 2016-08-11


Meetings MONDAYS on a 6-8 week basis for coffee/tea and a chat and to discuss ideas for future meetings and support

2016 Meetings

Dates: 3rd October 2016, 5th December 2016

Time: 10.30am – 12.45pm

Venue: Cambridge Cancer Help Centre, David Rayner Building, Scotsdales Garden Centre, 120 Shelford Road, Cambridge CB22 5JT (free parking)


Please contact Caroline Fookes (carfoo@waitrose.com), or Maria Piggin (contact@pnhuk.org), for further information

Date: 2016-08-05

Upcoming webinars on PNH - knowledge is power as they say!

Understanding the Complement System and the PNH Clone, https://www.pathlms.com/aamdsif/webinars/1021

PNH: Current Thinking on the Disease, Diagnosis, and Treatment, https://www.pathlms.com/aamdsif/webinars/1106

Advances in PNH Treatment: What’s on the Horizon, https://www.pathlms.com/aamdsif/webinars/1108.


Date: 2016-04-17

Learn about clinical research and its role in improving healthcare

When: Starts Monday 6 June 2016
Where: Free online

Following the success of last year’s free course from the NIHR Clinical Research Network, you can now sign up to their next course taking place this June. The course explores improvement in healthcare through clinical research. Please see: https://www.futurelearn.com/courses/clinical-research?mc_cid=cc30b35803&mc_eid=d0bb50ce88

Date: 2016-04-17

Public attitudes to commercial access to health data

When: 9.30am, Tuesday 19 April 2016
Where: The Wellcome Trust: 215 Euston Road Gibbs Building, London NW1 2BE

The Wellcome Trust is holding an event looking at public attitudes to commercial organisations having access to health data. The event is based on research that they have recently commissioned on the topic.  Please see: https://www.eventbrite.co.uk/e/public-attitudes-to-commercial-access-to-health-data-tickets-24253238088?mc_cid=cc30b35803&mc_eid=d0bb50ce88

Date: 2016-04-04


Rare Disease Day Receptions 

In honour of this year’s Rare Disease Day (29 February 2016), parliamentary receptions will be held and you can now register to attend these. 

Westminster Reception – hosted by Liz Kendall MP

When: 12.30pm to 3pm, Wednesday 2 March 2016
Where: House of Commons, Terrace Marquee – St Margarets Street London SW1A 0AA

To find out more about the event, or to register, please go to https://www.eventbrite.co.uk/e/house-of-commons-london-wednesday-2nd-march-1230pm-3pm-tickets-20695782643?mc_cid=bc5ad582a5&mc_eid=d0bb50ce88

Welsh Reception – hosted by Darren Millar AM

When: 6pm to 8pm, Tuesday 23 February 2016
Where: National Assembly for Wales - The Senedd Cardiff, South Glamorgan CF99 1NA

To find out more about the event, or to register, please go to https://www.eventbrite.co.uk/e/welsh-assembly-cardiff-tuesday-23rd-february-6pm-8pm-tickets-20696441614?mc_cid=bc5ad582a5&mc_eid=d0bb50ce88

If you would like to know more about Rare Diseases Day 2016, please see http://www.rarediseaseday.org.


Date: 2016-04-01

Project on Coping with Pain and Fatigue

One of the members of our group is looking for volunteers who are patients to take part in a project she is doing as a part of her PhD at SLADE School of Fine Art/UCL about coping with pain and fatigue. 

She is interested in the phenomenon of a human body as a repository for memories, in order to reconstruct and show methods of dealing with pain and loss, linguistic barriers and inexpressibility of emotions. Her project addresses the issue of the representation of physical and emotional crisis with special attention to the self-soothing and self-analysis processes, which are both relevant stages of dealing with a critical situation. 

Over the next six months, she plans to interview and analyse data from volunteers with chronic health conditions who live in pain and struggle with fatigue and at the same time she hopes to prepare a photographic series and videos on the problems we encounter while carrying out conversations and how we tackled those problems. An example of photos are attached. She is planning to visit Portsmouth, Brighton, Liverpool, York, Bristol, Bath, Birmingham and Manchester however she is also open to visiting volunteers in other places too. 

If you are interested or would like to know more, please contact us on contact@pnhuk.org and we will put you in touch with her.


Date: 2016-04-01

Save the Date - PNH Patient and Family Conference - Saturday 25 June 2016

PNH Support is organising a free one day PNH Patient and Family Conference on Saturday 25th June 2016 at a central London location to be confirmed. We anticipate that the conference will start in the late morning (approximately 11am) to enable people to travel from outside London on the same day and will finish at approximately 4.30pm. The conference will be an opportunity for patients and their families from around the country to meet one another and hear from our expert clinicians about PNH, any relevant developments in the disease/its treatment as well as ask questions. We hope to also have some sessions about non-clinical subjects, for example, managing fatigue, diet etc. There will also be plenty of opportunity to socialise and meet one another. Please do let us know if you have a suggestion for a session that you would like to see addressed/covered at this conference either by the clinicians or otherwise. We are aware that London may not be a convenient venue for everyone to attend a conference however we are endeavouring to accommodate as many people as possible. We are able to assist with travel expenses where necessary so please do contact us if you need assistance with these. An agenda for the conference will be circulated in due course.


We also intend to hold the inauguaral Annual General Meeting (AGM) for the members of PNH Support on the same day before the conference begins at approximately 10am (time to be confirmed). This will last about one hour (maximum). We welcome all members to attend this meeting and an agenda will be circulated closer to the time.


Should you have any questions/suggestions about the conference or AGM or membership of PNH Support, please contact us on contact@pnhuk.org.



Date: 2016-01-14

Webinar Explaining Drug Repurposing - 16 February 2016, 11am to 12.30pm

Findacure’s has teamed up with Healx, a Cambridge based social venture, to offer our first joint Drug Repurposing webinar.  

Join Dr David Cavalla from Healx and Dr Rick Thompson from Findacure, who will share with you their knowledge and experience of drug repurposing and discover how it can help the rare disease community.

With conventional drug discovery being a lengthy and costly process, there is now much discussion about drug repurposing, and how it can bring more effective and cheaper treatments to those living with a rare disease.

The 60 minute live webinar offers a comprehensive introduction to drug repurposing and aims to help you understand the process involved in repurposing drugs and what benefits this can bring. There will also be the opportunity for questions with both Rick and David following the end of the presentations.

Please click here to register https://www.eventbrite.co.uk/e/drug-repurposing-explained-webinar-tickets-20696173813?aff=es2


Date: 2016-01-13

Findacure Scientific Conference 

When: Monday 29 February 2016

Where: The Royal Institution of Great Britain

Findacure's annual scientific conference will look at drug repurposing for rare diseases. The conference will bring together patient groups, clinicians and industry to look at the potential benefits of drug repurposing as a mechanism for rare disease patients to access treatments as quickly as possible.

To find out more about this event and register to attend see https://www.eventbrite.co.uk/e/findacure-scientific-conference-drug-repurposing-for-rare-diseases-tickets-19442503050?mc_cid=bc5ad582a5&mc_eid=d0bb50ce88

Date: 2015-11-09

Research to be Presented at the American Society of Hematology (ASH) 2015 Annual Meeting (5 to 8 December 2015 in Florida, USA)

The following research abstracts will be presented in a poster session on Monday, December 7, 2015, from 6:00 p.m. to 8:00 p.m., Eastern Standard Time (EST):

  • Abstract 3339: “Patients with Paroxysmal Nocturnal Hemoglobinuria and Hemolysis Demonstrate More Frequent Disease-Related Features Than Those without Hemolysis, but Similar Proportions Experience Thromboembolism,” Yeneral, et al.

    Accessible at: https://ash.confex.com/ash/2015/webprogram/Paper79977.html

Date: 2015-11-04

Rare Disease Day Reception Dates Released

There will be parliamentary receptions to mark Rare Disease Day 2016 as follows. More details of how to register will follow.

  • Welsh Reception, 23rd February, 6-8pm, Senedd Cardiff.
  • Scottish Reception, Tuesday 1st March, Holyrood, Edinburgh.
  • Westminster Reception, Wednesday 2nd March, Houses of Parliament, London.

Date: 2015-11-04

Live Presentation from Dr Lawrence Rice on PNH - 14 November 2015

The Aplastic Anaemia & MDS International Foundation (AAMDSIF) is hosting their next patient and family conference in Tampa, Florida, USA on 14 November 2015 and Dr. Lawrence Rice (Haematologist) will be presenting on PNH.  His session will be able to be accessed live so patients and families from anywhere in the world can participate from their homes.  Registration is required.  To register, please go to:  http://webcast.eventtechnologysolutions.com/customer/aamds/2015/11-03/registration.asp


Date: 2015-10-15


‘Our Vision’ – An opportunity to help shape the future direction for nurses, midwives and care staff

NHS England is asking patients and carers, and organisations advocating on behalf of patients/carers, to give their views on what should be included in NHS England’s new strategy for nurses, midwives and care staff. The strategy is called ‘Our Vision’. You can get involved by:

  • attending a workshop in Leeds on 2 November 2015. Please email england.ourvision@nhs.net by 29 October 2015; or call Joe on 0113 825 2979 for further information;

  • contacting them directly using the details above or writing to: Our Vision, Nursing Division, Nursing Directorate, 5W52, NHS England, Quarry House, Leeds LS2 7UE; or tweeting @6CsLive using the hashtag #ourvision.

Date: 2015-10-15


The NHS Citizen Assembly – 25 November 2015

The NHS Citizen Assembly is taking place in London on 25 November 2015. Over the past few months, citizens from across England have been using face-to-face meetings and the NHS Citizen online forum to raise issues and tell us how they want the NHS to evolve. The Assembly will bring together the NHS England Board, policy-makers and citizens to:

  • explore the five issues raised by citizens;

  • enable participants to work with senior NHS managers to create solutions;

  • enable Board members to listen to participants’ concerns and proposals, and make commitments on next steps; and

  • provide an update on progress.

Date: 2015-10-07


Northern Ireland Rare Disease Partnership (NIRDP) family fun day and AGM

When: Saturday 24 October 2015, 1.30pm - 4pm
Where: Maldron Hotel, Belfast International Airport

There’ll be flower arranging, face painting, and a raffle. The NIRDP AGM will be held from 2pm to 2.30pm where you can find out what happened last year and look ahead to what is coming up in the year ahead. To register, click the following link:


Date: 2015-09-23

PNH National Service Patient Support Meeting - 30 September 2015

The PNH National Service at Kings College Hospital is holding a patient support meeting on Wednesday 30th September 2015 at 4pm until 7pm at Siobhan Davies Studios, 85 St George's Road, London, SE1 6ER. Come along if you can and meet other patients! If you can't make the whole meeting, please just come for as long as you can.

Date: 2015-09-23

The European Organisation for Rare Disease (EURORDIS) has announced that the 2016 Rare Disease Day theme and slogan.

Theme: Patient Voice
Slogan: Join us in making the voice of rare diseases heard

Rare Disease Day will be held on Monday, 29 February 2016, you can find more details about this herehttp://www.eurordis.org/content/rare-disease-day-2016-theme-and-slogan

Date: 2015-09-08

News from Wales  

Patient and Public Involvement Group (PAPIG) meeting

When: 9am Tuesday 22 September 2015
Where: Academic Centre at Llandough Hospital

On the agenda:
  • the All Wales Medicines Strategy Group (AWMSG) and the All Wales Therapeutics and Toxicology Committee (AWTTC)’s role in the upcoming patient and public involvement group meeting
  • update about the role of the Clinical and Patient Involvement Group (CAPIG) as part of the new orphan and ultra-orphan appraisal process
  • information about the upcoming Citizen's Jury that is being developed to reduce antibiotic prescribing in Wales
  • the role of the patient voice in medicine appraisals and reporting adverse drug reaction
For more information or to register for the meeting, please contact claire.davis@wales.nhs.uk -  AWTTC Senior Appraisal Scientist. 
Stratified Medicines in Wales 

When: Tuesday, 29th September 2015, 12.30pm – 1.15pm
Where: Media Briefing Room – Senedd, Cardiff Bay​

You are invited to join Genetic Alliance UK, ​the Association of the British Pharmaceutical Industry (ABPI) Cymru Wales and ​Cancer Research UK at a joint-meeting, sponsored by David Rees AM.

The aim of this meeting is to discuss the role and use of stratified medicines in Wales and the potential of a multi-stakeholder strategic review during the coming years to identify key challenges and further policy measures that would improve patients’ access to these innovative treatments. Speaking at this event will be Dr Rachel Butler, FRCPath, Head of All Wales Genetics Laboratory, and Dr Virginia Acha, Director of Research, Medical and Innovation, ABPI.

If you would be interested in joining in on the day, please email rhodri@positifgroup.co.uk.


Rare Disease Patient Network Launch Event

When: 20th October 2015
Where:​ The Hub, Cardiff Bay
The rare disease network has been formed to engage patients, families and patient organisation representatives in the work that Rare Disease UK is undertaking in Wales, in particular to support the implementation of the Welsh Rare Disease Plan that was published earlier this year. 
Topics for the day will include an introduction to clinical trials, new sequencing technologies and what they mean for patients with rare conditions. We will also be hearing from patients and carers about their experiences of living with rare conditions.
The keynote speaker will be Professor Chris Oliver from Birmingham University.
Refreshments will be provided throughout the day. Please contact Genetic Alliance's Development Officer in Wales, emma@geneticalliance.co.uk, for further information and a full agenda.


Date: 2015-08-24


Cambridge Rare Disease Summit

When: Monday 14 September 2015
Where: Cambridge Judge Business School, Trumpington Street, Cambridge, CB2 1AG


150 key stakeholders will come together to debate how to best address the huge social and therapeutic unmet needs around rare diseases. They want as many patient groups to be involved as possible! There will be an exciting line-up of speakers who are leaders in their fields, including a keynote video message from Prof Stephen Hawking.

Please see here for more details:

Date: 2015-08-13


Educating for Patient-Centred Care - UCLPartners Education Conference - 13 October 2015

Held on 13 October at the Royal College of General Practitioners, this conference is an opportunity to hear from some great patient and carer speakers, facilitators and panellists. There will be speakers and workshops about all aspects of education around patient centred care, from good practice sharing in particular specialties, to newer models of working and training. To register for one of the 40 free places for patients and carers, please contact fiona.mckenzie@uclpartners.com.

Date: 2015-06-30


The Rare Project

The Rare Project is a photography project designed to raise awareness of rare conditions whilst exploring attitudes towards those with a disability. It is designed to challenge preconceptions and to look beyond the outward appearance.

By the very nature of a syndrome or disease being rare it means it is rarely acknowledged or in the public eye. As awareness around a condition grows so too does acceptance. The aim of this project is to challenge the way the public see disability, whilst also informing people about different conditions.

The exhibition will include photographic portraits of adults and children with rare conditions doing something they have a passion for. Each image will be accompanied by information about that individual and a link to a website page where you can read more about the condition and linking to organisations representing those conditions.

To get involved in this project, or for more information please contact Ceridwen Hughes on 07971 983028. The website link is here: http://www.samebutdifferentcic.org.uk.

Date: 2015-06-23

Patient & Family Conferences - Living with Aplastic Anemia, MDA or PNH – 14 November 2015 – Tampa, Florida


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