NAITBABIES ANNUAL REPORT 2023
News and events from
1 June 2022 – 31 May 2023
Fetal and neonatal alloimmune thrombocytopenia - FNAIT
Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.
Reference and administrative details of the charity and its Trustees
Charity name: Naitbabies Other names we are known by: NAIT babies Charity number: 1161698 Principle address: 13 Redinnick Terrace Penzance Cornwall, TR18 4HR, England
Members of the CIO are also the trustees who administer the charity. Thea Palmer Chair Michelle Minshall Treasurer Stacy Corke Company Secretary Andrea Palmer Trustee
Treasurer : Michelle Minshall Minshall & Co Bridge House Nantwich Cheshire, CW5 7JX
Independent examiner: Mr Neill Hallam FCCA Crane and Johnston Chartered Certified Accountants 11 Alverton Terrace Penzance Cornwall, TR18 4JH
Bankers : HSBC, 1 Green Market Penzance Cornwall, TR18 2SD
Type of governing document : Constitution
How the charity is constituted : Charitable Incorporated Organisation (CIO) Foundation Date of governing document : 15 May 2015
Website :
www.naitbabies.org
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Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.
Naitbabies are members of: Genetic Alliance UK
Rare Disease UK
Mission Statement
Our mission is to be a strong voice for parents, children and families who have been diagnosed with or suspect that they may have Fetal and neonatal alloimmune thrombocytopenia - FNAIT.
Objectives and Activities
The objects of the charity as set out in the CIO’s constitution are:
The relief of sickness and the preservation and protection of good health of parents, children and their families and carers who have or are affected by the severe bleeding disorder neonatal alloimmune thrombocytopenia – FNAIT; in particular by the provision of support, advocacy and practical assistance as the trustees shall think fit;
To carry out, or to provide funds to support research into FNAIT, its causes, treatment and prevention and publish the useful results of such research; and
To advance the education of the public, in particular those involved in the diagnosis, treatment and care of those suffering from FNAIT.
Summary of the main activities undertaken for the public benefit in relation to these objects.
Naitbabies are a small Charitable Incorporated Organisation run by families whose children have been affected by the severe bleeding disorder FNAIT – Fetal and neonatal alloimmune thrombocytopenia.
Registered in the UK we are the only organisation for this disorder. We provide information to the public in general.
We run an international FNAIT Parents Support Group and advocate for parents worldwide. To date we have over 1,200 members.
We support research into FNAIT and have contact with an expert international medical panel of medical doctors who have a special interest in this devastating disease.
Watch our FNAIT video here
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Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.
What is FNAIT?
Fetal and neonatal alloimmune thrombocytopenia FNAIT (NAIT FMAIT) results from incompatibility between mother and baby for platelet-specific antigens that have been inherited from the father and are absent in mother.
The maternal immune response may make antibodies to destroy fetal platelets which she sees as foreign. Platelet destruction may cause bleeding into all major organs e.g. the stomach, spinal cord and lungs. The most feared is bleeding into the brain known as intracranial haemorrhage - ICH. Babies are at serious risk of death or permanent neurological impairment such as epilepsy, cortical blindness, cerebral palsy, precocious puberty, motor and cognitive delays and sensory processing disorders.
3 Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.
NAIT is seen as the platelet equivalent of the red blood cell disease Hemolytic Disease of the Fetus and Newborn - HDFN more commonly known as Rhesus disease.
Routine antenatal care has included screening of all expectant mothers for HDFN since the early 1970’s. No country carries out antenatal screening for NAIT although it has been known about since the 1950’s and is very well documented. Rarer diseases are also screened for.
Treatment is available for mothers who have developed antibodies against their unborn baby’s platelets and the success rate is high. If subsequent pregnancies are not treated they may also be at risk.
Platelets forming in bone marrow
Highlights of our journey through the last year.
June 2022
NORD. On the 21 June 2022 Naitbabies were invited to register as a patient organisation on the North American Dare Disease Organization Database. https://rarediseases.org/non-member-patient/naitbabies/
July
On July 2 Alan Halliday and his team mates Gareth Rees, Garry Bowman, Andy Donald, James Phillips, Andy Hogg, James Turner, Jeff Biggs and Nathan Turner took part in the UK Yorkshire Three Peaks Challenge a circular trek which encompasses three major peaks in the Yorkshire
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Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.
Dales, Pen-Y-Ghent, Whernside, and Ingleborough in under 12 hours raising a magnificent £1,473.16.
Alan’s 8 year old son Theo was affected by FNAIT after being born with a platelet count of only 11k. He received blood, IVIG and platelet transfusions all within the first 18 hours of his birth.
On July 9-13 The International Society on Thrombosis and Haemostasis (ISTH) reconvened for the ISTH 2022 Congress held in London, England. https://www.isth2022.org/ Professor Ulrich Sachs Germany, presented the manuscript PB1110 - Buffy - coat - derived megakaryocytes for monoclonal antibody immobilization of platelet antigens ( MAIMA ) assay .
August
' He's never alone'
One of our FNAIT Parents Support Group members, Carlos Marquez from New Jersey, USA, has a tattoo for each of his 5 children. This one, which depicts a shunt and the words ‘’His fight is my fight’’ is EXTRA special as his son Luke has already been through five brain surgeries due to intracranial haemorrhages caused by FNAIT and has a shunt fitted into his brain.
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Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.
A shunt is a hollow tube surgically placed in the brain to help drain cerebrospinal fluid and redirect it to another location in the body where it can be reabsorbed.
September
September 28, 2022
Preliminary data from the ongoing Phase 1b study shows RLYB212 rapidly and completely eliminates transfused HPA-1a positive platelets. More news is expected in the first quarter of 2023.
Rallybio Announces Positive Preliminary Results for RLYB212, an anti-HPA-1a Monoclonal Antibody for the Prevention of Fetal and Neonatal Alloimmune Thrombocytopenia
October
FNAIT SURVEY.
Naitbabies have been collaborating with Dr J B Bussel, Weill Cornell Medical University on a questionnaire for women who have fetal and neonatal alloimmune thrombocytopenia. This research project was started in 2020 and delayed for 2 years due to the Covid pandemic.
November
AGM – 28 November 2022
December
Grant. In December we were delighted to be able to support Dr T W de Vos, Leiden Medical University by providing a grant of £1000 towards his important FNAIT doctorate thesis publication.
January 2023
31 Jan – FNAIT Survey completed. The results will be published in 2023.
February
28 February Rare Disease Day
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Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.
March
Latest news on the development of a prophylactic treatment for FNAIT.
6 March 2023, Rallybio Announced Clinical Proof-of-Concept Achieved for its Lead Product Candidate, RLYB212, a Monoclonal Anti-HPA-1a Antibody, in the First Quarter of 2023. https://investors.rallybio.com/news-releases/news-release-details/rallybio-announces-proofconcept-achieved-rlyb212-novel
The Company also announced today that RLYB211, a plasma-derived polyclonal anti-HPA-1a antibody, will not be advanced further in clinical development.
25 – 31 March – FNAIT Awareness Week.
Our awareness week competition was won this year by one of our FNAIT families living in Northern Ireland.
April
On April 13th and 14th, Leiden University Medical Center together with Sanquin (Sanquin Research and Diagnostic) Services, Holland, organized the 𝐈𝐧𝐭𝐞𝐫𝐧𝐚𝐭𝐢𝐨𝐧𝐚𝐥 𝐂𝐨𝐧𝐬𝐞𝐧𝐬𝐮𝐬 𝐌𝐞𝐞𝐭𝐢𝐧𝐠 𝐨𝐧 𝐅𝐞𝐭𝐚𝐥 𝐚𝐧𝐝 𝐍𝐞𝐨𝐧𝐚𝐭𝐚𝐥 𝐀𝐥𝐥𝐨𝐢𝐦𝐦𝐮𝐧𝐞 𝐓𝐡𝐫𝐨𝐦𝐛𝐨𝐜𝐲𝐭𝐨𝐩𝐞𝐧𝐢𝐚: 𝐅𝐍𝐀𝐈𝐓.
After the conference a spokesman for LUMC posted ‘’We were pleased to welcome so many colleagues from various countries with expertise in fetal maternal medicine, in neonatology, in laboratory testing, in basic science and representatives from NAITbabies, who have direct personal experience in how FNAIT itself can impact the life of children and their families’’.
May
Support group Membership = 1,611 as of May 29 2023.
Naitbabies CIO run a private support group for FNAIT parents with members from all over the world.
May 31 2023 - End of Year
Many thanks to everyone for reading our review and for your continued support!!!!!
Naitbabies.org
7 Charitable Incorporated Organisation (Foundation}. Registered in England and Wales No.1161698.
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Naitbables.org Notes to the Accounts for the year ended 31 May 2023 1 Accountlng pollcles AccountiTng Convention The accounts have been prepared on the reip1S and paymenls basis. The accounts are in accordance with applicable accounting standards and comply wilh the Charities IAccounls and Reports) Regulations issued under the Charities Act 2011. Recelpts Receipts are the total amounts received by the CIO for goods and services provided lo the public. Any donations have been included in the accounts when received. Payments Expendllure is included on a payments basis, inolusive of any VAT which cannot be recovered. 2 Funds 2023 Al 1 June 2022 Nel feceipls for the perlod 45,611 3.376 Al 31 May 2023 48,987 3 GuaranteDs The trustees confirm, in accordance wllh the Charitable Incorporated Organisalions IGenerall Regulations 2012, that al the year ending the CIO did not have any outstanding guarantees lo third parties nor any debts secured on assets ol the CIO. 4 Trusteos None of the trustees lor any persons connected with them) received any remuneration during the period. None of the Iruslees We relmbursed for any expenses durlng the perlod,