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https://www.rd-rp.com/uk-gdpr-data-protection-and-privacy-policy daily 0.75 2026-01-26 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/c25ea5a9-1399-4de9-9754-2788df855ee6/Policy+PDF+Image.png https://www.rd-rp.com/data-retention-and-disposal-policy daily 0.75 2026-01-26 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/6872cd8a-d03f-4eaf-998f-b34a7426b7b6/Data+retention.png Data Retention and Disposal Policy https://www.rd-rp.com/publications daily 0.75 2026-04-08 https://www.rd-rp.com/publications/posters monthly 0.5 2026-04-08 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/f3d90972-40f0-48cc-a17f-465916fb1794/Supporting+shared+decision-making+in+Pompe+disease.png Publications - Posters Supporting shared decision-making in Pompe disease: Making complex treatment evidence accessible for patients. POMPE DISEASE | 02/2026. Poster presented at WORLD Symposium February 2026, San Diego https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/803c258c-ba35-4a04-8de6-7183259a0448/Insights+from+Patients+%281%29.png Publications - Posters Empowering Fabry Conversations: Bridging the Communication Gaps. FABRY | 02/2026. Poster presented at WORLD Symposium February 2026, San Diego https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/237f6440-d987-4e40-9918-1e3c13d4d98d/2026+World+2026+poster.png Publications - Posters Insights from Patients, Caregivers and Healthcare Professionals on the Challenges of Lysosomal Disorders. LYSOSOMAL DISORDERS | 02/2026. Poster presented at WORLD Symposium February 2026, San Diego https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/0ad80c9b-83d9-441b-8683-cb90cc8dfcc2/1.+Barriers+and+facilitators.png Publications - Posters Barriers and facilitators to clinical trial participation: improving accessibility, logistics and awareness. FABRY | 02/2025. Poster presented at WORLD Symposium February 2025, San Diego https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/25fb0caf-965d-4e70-9a44-2be14169d88c/2.+Medical+educational+needs+-+Chiesi.png Publications - Posters Medical Education Needs to Improve Diagnosis of Fabry Disease in the UK. FABRY | 01/2024. Poster presented at WORLD Symposium February 2024, San Diego https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/29c0ebc1-a880-40d2-9bef-1ac5a97b2dfe/3.+WORLD+2024+Fabry+QoL+and+mental+health+e+poster.png Publications - Posters The impact of Fabry symptoms on patients’ quality of life (QoL) and mental health - a qualitative interview study in the UK. FABRY | 02/2024. Poster presented at WORLD Symposium February 2024, San Diego https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/8fef8056-00c8-4f52-8fbb-6893e37dac96/4.+Barriers+clinical+trials.png Publications - Posters Barriers to Participation in Clinical Trials. RARE DISEASES | 02/2022. Poster presented at WORLD Symposium February 2022, Orlando https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/d67d7468-3c9d-4442-b725-39fe6a110e0f/5.++Mortality+alpha-mann.png Publications - Posters Mortality in patients with Alpha-mannosidosis. ALPHA-MANNOSIDOSIS | 03/2022. Poster presented at WORLD Symposium February 2022, Orlando https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/7f49a900-7282-4e75-bae9-9e4cf9128f51/6.+Importance+MLD.png Publications - Posters The importance of early diagnosis and views on newborn screening in Metachromatic Leukodystrophy. MLD | 03/2022. Poster presented at WORLD Symposium February 2022, Orlando https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/6d93bc00-7a1f-4d70-a012-0fa492dcc1c0/7.+MLD+burden.png Publications - Posters Metachromatic leukodystrophy burden of disease. MLD | 03/2022. Poster presented at WORLD Symposium February 2023, San Diego https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/9055d1e6-448b-4952-b9dd-693aeb73d68e/8.+Diagnosis+Fabry.png Publications - Posters Diagnosis of Fabry disease in the UK. FABRY | 03/2022 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/1017733b-6b4c-4c16-9628-d77e102f8dbe/9.+Covid-19+ERT.png Publications - Posters The impact of COVID-19 on Fabry patients receiving enzyme replacement therapy (ERT). FABRY | 03/2022 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/8d682bd0-00de-424f-8838-2d488528ba12/10.+Early+indicators+MPS.png Publications - Posters Identifying early indicators of mucopolysaccharidosis disorders using UK parent-held child health records. MPS (ALL) | 07/2021. Poster presented at 16th International Symposium on MPS and Related Diseases July 2021, Barcelona https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/9facf0ea-81cd-4514-858f-8dfb572d57ea/11.+Challenges+ultra-rare.png Publications - Posters Understanding challenges for ultra-rare lysosomal storage disorders: Patient and caregiver experience of care and support through the disease journey. ULTRA-RARE LYSOSOMAL STORAGE DISORDERS | 03/2021. Poster presented at WORLD Symposium February 2021, Virtual https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/0bb6d588-c27c-4245-859a-981c61859d02/12.+Clin+trials+covid-19.png Publications - Posters Maintaining access to clinical trials during the COVID-19 pandemic. RARE DISEASES | 03/2021. Poster presented at WORLD Symposium February 2021, Virtual https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/2bdf52f7-257e-4412-a402-2f10ccd4cd25/13.+Diagnosis+MPS+UK.png Publications - Posters Diagnosis of mucopolysaccharidoses in the UK. MPS (ALL) | 03/2021 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/a3abe3ba-7c17-4824-84d7-a9b5fd7b2d5c/16.+Intestinal+disease+MPS+IIIA.png Publications - Posters Prevalence of intestinal disease as terminal even in Mucopolysaccharidosis Type III – A study of 136 deceased patients. MPS III | 02/2020. Poster presented at WORLD Symposium February 2020, Orlando https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/beb16ab2-7678-44a4-a716-f36727dc6c03/14.+Fabry+Families.png Publications - Posters Understanding Fabry in Families Study – the availability of pedigree testing, genetic counselling and understanding of inheritance across Fabry International Network countries. FABRY | 02/2020. Poster presented at WORLD Symposium February 2020, Orlando https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/fdffe0a7-c42e-4de3-be7c-b31d337f8697/15.+Supporting+adults+MPS.png Publications - Posters Supporting adults living with mucopolysaccharide (MPS) diseases: Understanding current experiences and future challenges. MPS (ALL) | 02/2020. Poster presented at WORLD Symposium February 2020, Orlando https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/4b3833e6-80a0-48ea-a41f-c1efa4e863c1/17.+Audit+clin+trials.png Publications - Posters Audit of a rare disease clinical trial support service. RARE DISEASES | 01/2020. Poster presented at WORLD Symposium February 2020, Orlando https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/b3156bb6-119d-4e3b-bba1-bc0100fd3fae/18.+Disease+Burden+Pompe.png Publications - Posters Disease Burden and Unmet Needs: Results from a New Survey in Adult Patients Receiving Enzyme Replacement Therapy for Pompe Disease in the United Kingdom. POMPE DISEASE | 09/2019. Poster presented at Muscle Study Group Annual Scientific Meeting September 2019, Snowbird https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/776a53ee-2e55-483b-b2b5-3c9b7affb55f/19.+Burden+of+illness+MPS+III.png Publications - Posters Burden of illness in Sanfilippo disease (MPS III) - results from and international caregiver survey. MPS III | 03/2019. Poster presented at WORLD Symposium February 2019, Orlando https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/e41f5941-e355-474d-ba51-8d9626a52a48/20.+Pathway+to+diagnosis+MPS+III.png Publications - Posters Pathway to diagnosis in Sanfilippo disease (MPS III) – results from an international caregiver survey. MPS III | 03/2019. Poster presented at WORLD Symposium February 2019, Orlando https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/e7469f53-2a8e-4967-9e67-65c173c45d0a/21.+Fabry+in+families.png Publications - Posters Understanding Fabry in families: Preliminary findings from a global survey. FABRY | 03/2019. Poster presented at WORLD Symposium February 2019, Orlando https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/65a631a9-3801-4edb-9f59-13e549ea49b9/22.+Patient+recorded+outcomes+MPS+IVA.png Publications - Posters Patient reported outcomes in MPS IVA patients receiving enzyme replacement therapy. MPS IVA | 03/2019. Poster presented at WORLD Symposium February 2019, Orlando https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/da79056d-dd0a-496d-96de-9d1e9941e03c/23.+Diagnosing+ultra-rare.png Publications - Posters The challenges of diagnosing patients with an ultra-rare disease – insights from the European MPS VII study. MPSVII | 09/2018. Poster presented at The 3rd International Conference on Pediatrics and Pediatric Surgery May 2018, Frankfurt https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/6a771d5c-957f-4dc4-83f5-e55543315094/24.++Diagonsis+disease+burden+MPS+VII.png Publications - Posters Diagnosis and disease burden of MPS VII – a European survey. MPS VII | 09/2018. Poster presented at The 15th International Symposium of the International MPS Network August 2018, San Diego and Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium September 2018, Athens https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/ffdc7274-793d-4995-8c2f-6e31a86d0205/25.+Patient+orgnasiations+MPS+III.png Publications - Posters Patient organisations working in partnership to research the patient experience of rare diseases – the Sanfilippo (MPS III) survey. MPS III | 03/2018. Poster presented at WORLD Symposium February 2018, San Diego https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/ad69fea1-d7d4-4f51-bad9-524a7314bb98/26.+Elosulphase+alfa+Morquio+A.png Publications - Posters Impact of elosulfase alfa treatment on patient-reported outcomes in Morquio A Syndrome. MPS IVA | 09/2017. Poster presented at 13th International Congress of Inborn Errors of Metabolism (ICIEM) September 2017, Rio de Janeiro https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/d600585a-b9b7-4cda-938d-de466fc24c60/27.+Educational+journey+Morquio.png Publications - Posters The educational journey of individuals with MPS IVA Morquio Disease. MPS IVA | 08/2016. Poster presented at The 14th International MPS Symposium July 2016, Bonn https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/d8945b2d-70cf-4443-9feb-915d03c59633/28.+Diagnosis+and+treatment+Hunter.png Publications - Posters Diagnosis and treatment of individuals with MPS II Hunter disease in the UK. MPS II | 08/2016. Poster presented at The 14th International MPS Symposium July 2016, Bonn https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/7eed6e11-a2de-46e6-9f7a-8938587fce89/29.+Educational+journey+Hunter.png Publications - Posters The educational journey of individuals with MPS II Hunter Disease in the UK. MPS II | 08/2016. Poster presented at The 14th International MPS Symposium July 2016, Bonn https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/25ac05a6-e19d-4b39-80f5-e8373459f170/30.+Multi+stakeholder+Morquio.png Publications - Posters Multi-stakeholder engagement leading to access to treatment for MPS IVA (Morquio A) – a model for the ultra-rare disease community. MPS IVA | 07/2016. Poster presented at The 14th International MPS Symposium July 2016, Bonn https://www.rd-rp.com/publications/booklets monthly 0.5 2026-02-25 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/894fe86b-cd7b-4be4-8db0-3c521bc44280/11.+Fabry+7.png Publications - Booklets Fabry Finings Issue 7. FABRY | Summer/2023 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/a5a35294-dd16-4ab6-b123-973ce3efe55c/10.+Fabry+6.png Publications - Booklets Fabry Findings. Issue 6. FABRY | Spring/2022 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/617549a2-81ba-4f06-9494-d6f62bf9fadd/1.+Fabry+5.PNG Publications - Booklets Fabry Findings Issue 5. FABRY | 10/2021 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/a6ea5612-be1c-48fe-bb85-bc4addf56b31/2.+Covid-19.PNG Publications - Booklets Expert guidance on Covid-19 for patients. LYSOSOMAL STORAGE DISEASES | 04/2021 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/c800a545-e94c-4103-8c06-d3dda6ba7435/3.+Focus+on+Fabry.PNG Publications - Booklets Focus on Fabry. Fabry treatment update. FABRY | 10/2020 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/99ace1f4-1ae1-4a82-b806-0e4073b12456/4.+Fabry+4.PNG Publications - Booklets Fabry Findings Issue 4. FABRY | 10/2020 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/76b0dc35-4bac-44bf-be85-4b9ff915018d/5.+Brineura.PNG Publications - Booklets Brineura treatment for CLN2 disease – The Managed Access Agreement Guide. CLN2 (BATTEN) | 07/2020 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/6b638897-29b4-4ee9-914f-91d8da9e5d31/6.+Fabry+3.PNG Publications - Booklets Fabry Findings Issue 3. FABRY | 03/2020 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/e1911d4c-51c7-4fe0-8896-33991fc105b9/7.+Fabry+2.PNG Publications - Booklets Fabry Findings Issue 2. FABRY | 12/2019 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/56cdca88-34ac-4c99-9e6f-d27b7990108b/8.+Vimizin.PNG Publications - Booklets Vimizim treatment for Morquio A – The Managed Access Agreement. MPS IVA | 06/2019 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/92764ddd-cf32-4bc3-bf8e-d9b414af2b4c/9.+Fabry+1.PNG Publications - Booklets Fabry Findings Issue 1. FABRY | 06/2019 https://www.rd-rp.com/publications/reports monthly 0.5 2026-02-25 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/b04a4c53-07f1-4b9d-9786-047d06a67a24/1.+MPS+Society+Vision.PNG Publications - Reports Defining the MPS Society vision. Findings from our stakeholder survey. RARE DISEASES | 07/2019 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/8510b473-7272-4aba-9668-e03d2b632da7/2.+Current+opportunites+Fabry.PNG Publications - Reports Clinical trials: Current opportunities in Fabry. FABRY | 03/2019 https://www.rd-rp.com/publications/manuscripts monthly 0.5 2026-02-25 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/1777039382216-VJJ6D8HBQJ1Q5FJUTYZ3/Supporting%2Bshared%2Bdecision-making%2Bin%2BPompe%2Bdisease%2B%25281%2529.png Publications - Manuscripts A communication toolkit for the most impactful symptoms of Fabry Disease: co-creation with Fabry disease patients and their treating clinicians in the UK. FABRY | NOV 2025 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/1726659616654-FD5O9I65241Q5L8SU9IP/Publication_18.09.2024.jpg Publications - Manuscripts Transition of patients with Gaucher disease type 1 from pediatric to adult care: results from two international surveys of patients and health care professionals. GAUCHER | AUG 2024 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/9de5fde3-b3e2-4484-b586-88a7e7320acf/ASMD.JPG Publications - Manuscripts Real‑life impacts of olipudase alfa: The experience of patients and families taking an enzyme replacement therapy for acid sphingomyelinase deficiency. ASMD | FEB 2024 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/5e6dbe92-a0ca-425f-a175-37e1a9daa471/0.+Burden+of+disease+MLD.PNG Publications - Manuscripts The burden of disease in metachromatic leukodystrophy: results of a caregiver survey in the UK and Republic of Ireland. MLD | FEB 2024 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/fecf48d3-fca0-4c1e-8d90-0fe7e4fda7ef/1.+MLD.PNG Publications - Manuscripts The importance of early diagnosis and views on newborn screening in metachromatic leukodystophy: results of a Caregiver Survey in the UK and Republic of Ireland. MLD | 02/2023 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/481edd73-f555-4f0b-ab5b-c7c68c79aa62/2.+Clinical+networks.PNG Publications - Manuscripts Enhancing the value of clinical networks for rare diseases. RARE DISEASES | APR 2022 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/3b8f7fd6-000d-494c-b0ac-383aeec04c87/3.+Psych+support.PNG Publications - Manuscripts The importance of psychological support for parents and caregivers of children with a rare disease diagnosis. RARE DISEASES | APR 2022 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/142e8be2-f881-4dec-82a6-5b3000587797/4.+Alpha-mann.PNG Publications - Manuscripts Mortality in patients with alpha‑mannosidosis: a review of patients’ data and the literature. ALPHA-MANNOSIDOSIS | MAR 2022 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/d008a177-9014-4997-bd63-08b2fef8da2d/5.+Covid-19.PNG Publications - Manuscripts Impact of the COVID‑19 pandemic on access to the cerliponase alfa managed access agreement in England for CLN2 treatment. CLN2 (BATTEN) | JAN 2022 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/f299293f-444f-4e14-9bc2-b1063e640dfa/6.+Elosulfase+alpha.PNG Publications - Manuscripts Elosulfase alfa in the treatment of mucopolysaccharidosis type IVA: insights from the first managed access agreement. MPS IVA SEPT 2021 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/5cb9cf4b-ce79-4a90-b56e-4f9407cf6502/7.+Gastro.PNG Publications - Manuscripts Gastrointestinal Manifestations in Mucopolysaccharidosis Type III: Review of Death Certificates and the Literature. MPS III | SEPT 2021 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/8b0fb1e1-3286-4dc8-812a-5d7705e0aa2e/8.++Elosulfase+alpha+long+term.PNG Publications - Manuscripts Impact of long‑term elosulfase alfa treatment on clinical and patient‑reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England. MPS IVA | JAN 2021 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/0b7bd8a9-899e-451a-a594-28c55efdfcc2/9.+Cancel+surveilance.PNG Publications - Manuscripts Cancer Surveillance Guideline for individuals with PTEN hamartoma tumour syndrome. PTEN HAMARTOMA TUMOUR SYNDROME | APR 2020 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/048a1c46-628e-4cd2-ac3c-2b934035dfe3/10.+Li-Fraumeni.PNG Publications - Manuscripts Guidelines for the Li–Fraumeni and heritable TP53-related cancer syndromes. LI-FRAUMENI | APR 2020 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/a0162080-23e5-438e-8ae3-883c4a577c15/11.+Burden+MPS+VII.PNG Publications - Manuscripts Pathway to diagnosis and burden of illness in MPS VII – a European caregiver survey. MPS VII | APR 2020 https://images.squarespace-cdn.com/content/v1/64e86ee094738235dc5f2a3b/b2d0ccc0-4922-46b9-bf10-7097b56b2071/12.+Diseas+progression+alpha-mann.PNG Publications - Manuscripts Disease progression of Alpha-mannosidosis – A UK natural history survey – manuscript. 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