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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 2026-02-25 https://www.rd-rp.com/publications/posters monthly 0.5 2026-02-25 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 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. FABRY | 02/2026 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 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 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 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 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 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 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 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/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 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 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 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 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/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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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 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/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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