On 1st April 2019 General Practitioners (GP’s) around the globe will be hanging up their stethoscopes and hitting the streets to march for the first ever All Fools Day March.

The march has been organised by a group of pioneering researchers and medics who have made a breakthrough discovery that the ‘patients were in fact right all along’. A recent paper published on PubMed set out the results from a 15 year research project, costing over one billion pounds, which concluded that at no time in history has civilisation experienced a pandemic of Munchhausen syndrome or Munchhausen by proxy (previously factitious disorder) – an illness in which a person fabricates illness for themselves or a dependent and puts them through unnecessary medical treatment. The study went on further to conclude that it found 99.87% of patients presenting in the consultation room were credible and educated individuals, who were highly informed and able to articulate their symptoms accurately.
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The study states that these problem patients were in fact found to be affected by one or more of over 7,000 rare diseases which it estimated accounts for some 350 million patients worldwide.

Dr. A (who preferred to remain anonymous), told us:

Newly qualified GP, Dr Newby, told us “I am thrilled to be part of this, first of it’s kind, march showing solidarity for rare disease patients. It is an exciting time to be entering the profession. We are at the dawn of the new era of advanced `believing and listening` medicine!"

The organisers hope to see millions take to the streets and are already setting their sights on planning the 2020 march. “With a century of attitude to overturn there is much work to do, but this study and march are an important first step. Next we will be recalling the following posters which are known to adorn many a GP waiting room walls – PLEASE DON’T CONFUSE YOUR GOOGLE SEARCH WITH MY MEDICAL DEGREE.

Instead they will be replaced with the following Medical Association approved poster.

I PROMISE NOT TO UNDERMINE YOUR LIFETIME OF LIVING WITH YOUR CONDITION BASED ON MY ONE HOUR MEDICAL SCHOOL LECTURE ON IT.

​Doctors and patients alike all agree – these are exciting times!

#InThisTogether
#PatientDoctorPartnership
#WeAreListening 
​#DawnOfANewEra

Further information
​Eurodis would like to thank you in advance for your participation. For more information, visit Eurodis

Last week I attended Findacure’s Drug Repurposing for Rare Diseases Conference at the RCN HQ in London. There were a wide of range speakers from patient representatives, medical professionals, a medical student, patient groups to pharma companies. This range of perspectives and some really inspiring collaborations highlighted what can be achieved by sharing and learning from the unique expertise and skills of each of these groups.

The UK’s first drug trial for Rett Syndrome
After the warm welcome from Rick Thompson, CEO of Findacure, we heard from Reverse Rett and Rett UK. Rachael Bloom Stevenson, CEO of Reverse Rett talked about Reverse Rett’s involvement in the first UK clinical trial for Rett Syndrome in 2017, which was launched at King’s College Hospital in London under Professor Paramala Santosh. Becky Jenner, CEO of Rett UK and parent to Rosie, spoke about her daughter’s experience of participating on the trial and the benefits that they personally have experienced from the trial.

Repurposing nitisinone in alkaptonuria
Professor Lakshminarayan Ranganath, Director of the National Alkaptonuria Centre (NAC), is a consultant at the Royal Liverpool Hospital, he talked about repurposing nitisinone in alkaptonuria patients.

Alkaptonuria (AKU) is an iconic autosomal recessive condition, caused by a faulty enzyme (homogentisate dioxygenase) in the tyrosine degradation pathway that results in a build up of homogentisic acid (HGA) in the body. This affects multiple systems in the body including the spine, joints, heart, ligaments, eyes and kidneys.

Nitisinone has been used in a related disorder, hereditary tyrosinaemia 1, as the standard of care for more than 20 years. Data collected from the NAC shows a beneficial effect of nitisinone in AKU.

Pharnext on Pleotherapy
Pharnext is an advanced-clinical-stage biopharmaceutical company. Rodolphe Hajj, Chief Pharmacology Officer and Xavier Paoli, Chief Commercial Officer at Pharnext, gave a joint talk about Pleotherapy™, a platform that systemises the identification and development of new synergistic combinations of repositioned drugs for diseases with high unmet medical needs. These new therapeutic entities are called PLEODRUG™ and they are expected to feature high levels of efficacy and safety due to being formulated with new, optimal, lower doses of their individual components, and they target simultaneously several disease pathways.

Winner of The Student Voice essay
We had the pleasure of hearing from Logan Williams, winner of The Student Voice essay competition. Logan is a final year medical student at The University of Auckland. The title of his essay Repurposing: a rare opportunity: a brief insight into how implicit bias towards biomedicine impacts the care received by patients with a rare illness. His essay focused on his experience of treating a five-year-old boy with worster drought syndrome and how he became inadvertently caught in the trap of focusing on the boy’s diagnosis rather than the patient.

After speaking to the boy’s mother, Logan searched to understand the origin of biases towards biomedicine, with himself and the medical system, believing it is imperative that patients and families are given a voice.

Incentivising the repurposing of off-patent medicines
Dr Pan Pantziarka spoke about his work as part of ReDo (Repurposing Drug in Oncology) and an AMRC led repurposing round table group, which aimed to facilitate the use of off-patent repurposed medicines within the NHS.

Idebenone: how repurposing this drug is helping Duchenne muscular dystrophy
Catherine Lawrence, Senior Medical Advisor UK at Santhera and Janet Bloor, parent and Duchenne Advocate, and Vice Chair at RACC, jointly discussed advocacy for Duchenne, the development of the drug Idebenone and the subsequent clinical trial.

Duchenne muscular dystrophy is a rare genetic muscle wasting life-limiting disease. Santhera was the first pharmaceutical company to address the hugely unmet needs of older non-ambulatory boys, and has been trialling Idebenone, originally developed for Alzheimer’s, as a respiratory drug for Duchenne.

Janet talked about how her son (who is now 25) and has been on this trial for 18 months. Janet is a strong advocate of having a  registry for your disease that is interconnected on a global scale.

A life with PNH: from isolation to treatment 
The last speaker of the day was Suzanne Morris a PNH Patient Representative. She was diagnosed as a child in the 1970s when there was very little help or support. It was incredibly moving to here Suzanne speak of living her life in secret for decades. After her diagnosis and in the following years she talked of the difficulty of living with PNH in a time when there was a lot of prejudice in society against blood diseases. Suzanne also discussed the lack of focus on the mental side of conditions and how this is not taken in to consideration. This led to a discussion about compulsory counselling and the benefits that this could potentially give to patients and their families.
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Suzanne featured in our RARE Blood issue read it here.