Keith Berelowitz founded trialport after patient experience revealed clinical trial recruitment barriers. https://ow.ly/PtSm50Z51IZ

#ICYMI The Undiagnosed Hackathon unites global experts to solve undiagnosed diseases where traditional medicine fails. https://rarerevolutionmagazine.com/rare-insider/solving-the-unsolvable-a-revolutionary-new-model-for-diagnosing-rare-diseases/

Stichting TAPS Support, founder, Stephanie Ernst shares their top three unmet needs for the TAPS community, exploring the deficit in large scale connected data and the growing need for standardised care and fetal specific hardware. https://ow.ly/gTUs50Z2FLe

Amy Gaviglio, Connetics Consulting, asks what industry partners need to understand about the families and groups carrying our screening systems forward, and the question we should all be willing to ask https://ow.ly/pBub50Z2Fxe

We asked our community "For healthcare professionals, how important are metrics when measuring the “success” of your Rare Disease Day activities?" Every week insights brings you the latest views from your global RARE community. https://ow.ly/Sqxq50Z11Ir  

Ivor Campbell, CEO of Snedden Campbell, reveals how AI is transforming senior specialist recruitment in life sciences, making 'one size fits all' outdated. https://rarerevolutionmagazine.com/rare-insider/the-failure-of-the-usual-suspects-approach-to-life-science-recruitment

Dr Tarek Ebrahim, a biotech leader, shows how rare disease leadership means letting patients shape care decisions. https://rarerevolutionmagazine.com/rare-insider/patient-centricity-is-the-mission-patient-inclusion-is-the-operating-model/

We asked our community "For healthcare professionals, did you see a measurable impact from your Rare Disease Day activities?" Every week insights brings you the latest views from your global RARE community.  Read more here: https://ow.ly/pw1a50YVY1Z

The BioForge is using its BioOptima AI platform to revolutionise bioprocess development, cutting the timeline for drug manufacturing scale-up from months to just weeks. Learn how this breakthrough is closing biotech’s "valley of death". Read the article here: https://ow.ly/qrUG50YVY0g

Tune in to "Rethinking standards of care in rare diseases: what we can learn from myasthenia gravis" on May 13th 2026 2pm BST. Register now: https://bit.ly/UCBWebinar-RethinkingMG

Meet Deborah O'Neil, CEO of NovaBiotics Ltd. She discusses her journey from academia to founding a biotech company focused on translating innovation into treatments for difficult infectious-inflammatory diseases, including cystic fibrosis. https://ow.ly/Rvp050YVXTG

We asked our community "How important are metrics when measuring the “success” of your Rare Disease Day activities?" Every week insights brings you the latest views from your global RARE community.  https://ow.ly/uimV50YUrTS

Simon Bull, shares their top three unmet needs for the Charcot-Marie-Tooth (CMT) community in the UK. Discover their main asks of industry, focusing on boosting healthcare professional awareness, expanding community outreach, and ensuring the charity's development. https://ow.ly/ZMNb50YUrTB

Toni Mathieson, Niemann-Pick UK and professor Pamela Kearns, University of Birmingham, discuss the need to involve patients in clinical trials from the earliest stages and outline the current R&D barriers faced by the NHS. Read here: https://ow.ly/r4sS50YUrSP

We asked our community "Do you find you have better engagement/successful campaigns on Rare Disease Day or on your awareness day?".  Every week insights brings you the latest views from your global RARE community.  https://ow.ly/eilQ50YOBCi

Despite falling participant numbers and rankings, Prof Raghib Ali is optimistic about the future of UK clinical trials. Learn how ambitious government targets and the unique data assets are driving opportunities for growth, faster trial set-up, and world-leading research. https://bit.ly/4ctj31i

Meet Gemma Gapper, a leader in pharmaceuticals and biotech with a 30-year career dedicated to patient access for rare conditions. Discover her journey and her insights on women's leadership in the field. https://bit.ly/4vTKTLR

Breaking news. Gene therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA) receives EMA CHMP positive opinion. Read more here: https://ow.ly/IGmt50YP0p1 #BreakingNews #News #SMA #SpinalMuscularAtrophy #EMA

We asked our community "Did your increased Rare Disease Day campaign engagement help you reach a different or new audience?".  Every week insights brings you the latest views from your global RARE community. https://bit.ly/4swB6sd

Dr Kingyin Lee at the MHRA, and Clive Collett at the Health Research Authority (HRA), discuss upcoming regulatory clinical trial changes to help place the UK as a leader in this space and provide greater clarity for participants https://bit.ly/3OjPh5Q

BridgeBio is focused on developing a daily oral therapy for achondroplasia as an alternative to thousands of injections. This programme explores improved functionality and quality of life for families with the condition. Read more here: https://bit.ly/3QmiSMr

#ICYMI Clinical trials for rare diseases face unique challenges, from limited patient pools to high operational costs. With 30% of patients dropping out and over 85% of trials failing to recruit on time, it's clear that patient experience must be prioritised. https://bit.ly/4t0fCVs

The NCBRS Worldwide Foundation outlines three vital asks for industry. Read Lee Reavey's full insights. https://rarerevolutionmagazine.com/rare-insider/putting-you-in-the-heart-of-the-rare-community-ncbrs-worldwide-foundation/

UK firm Vitaccess and the Myasthenia Gravis Foundation of America (MGFA) have partnered to create the comprehensive Vitaccess Real MG (VRMG) registry. This platform will track the long-term reality of living with myasthenia gravis. https://bit.ly/3Qc5Ocq

We asked our community "For advocates: did you see measurable impact from your Rare Disease Day activities?". Every week insights brings you the latest views from your global RARE community. https://bit.ly/4siDtP7 #RareInsights

Donna shares her journey from creative agency life to becoming a full-time caregiver for her children with EDS. Her experience fuelled the documentary Complicated and the launch of the non-profit ElevateRARE. https://bit.ly/4seYzxT

The UK excels at rare disease policy, but implementation often lags. Owen Marks highlights the "under-delivery" gap between strategy and patient access, using the Innovative Medicines Fund as a case study. https://bit.ly/4tL860X

The future of organ transplants is being rewritten! Scientists are pushing the boundaries of bioprinting with the ambitious goal of printing a functional kidney in just 24 hours. Could this be the breakthrough that transforms healthcare? https://bit.ly/4bRpJEK

A powerful story of parent‑led innovation: how the FOXG1 Research Foundation has moved from a single diagnosis to sponsoring its own international gene therapy trial—redefining what’s possible in rare disease drug development. https://bit.ly/3Pyt3xc

article challenges social media‑led youth advocacy, arguing that visibility alone is not representation. Drawing on real‑world examples, it makes the case for investing in community‑based action and integrating young people into accountable, long‑term advocacy structures. https://bit.ly/412kCg2

Discover how China’s DeepSeek-R1, a low-cost, open-weight AI model, is democratising high-end drug discovery. Learn what this means for accelerating research and driving innovation. https://rarerevolutionmagazine.com/rare-insider/what-ai-innovations-like-chinas-deepseek-could-mean-for-rare-diseases/

AML has long resisted cell therapy, but CERo Therapeutics is challenging convention with its novel CER-T platform. Director Brandon Cieniewicz discusses their unique approach targeting a hidden molecule and the promising early clinical signals from the CertainT-1 trial. https://bit.ly/412TVbf

Grab a sneak peek from our interview for the article eith Nicole Zeitzer Johnson, co-founder and executive director,FOXG1 Research Foundation https://rarerevolutionmagazine.com/rare-insider/nicole-zeitzer-johnson-on-parent-led-drug-development-for-foxg1 #FOXG1

Is your health advice coming from AI? Our latest article dives into the impact of Google's AI Overview on accessing medical information, examining its accuracy, and the risks of misinformation. Learn why scientific organisations must adapt to this new era of dAIgnosis. https://bit.ly/4lF9j6V

Meet Elizabeth White, COO of Orphan Therapeutics Accelerator, championing drug development and access for ultra-rare diseases. Discover her insights on opportunities and barriers for women in biopharma https://bit.ly/3NupNlS

We asked our community "Do you have a clear understanding of how to compliantly raise awareness for study/trial awareness to the public?".  Every week insights brings you the latest views from your global RARE community. https://bit.ly/4dn54dZ

Grab a sneak peak at our interview with Brandon Cieniewicz, director of CERo Therapeutics on treating acute myloid leukaemia through targeting CER-T cells. https://bit.ly/4sHx2FL #AML #AcureMyloidLeukaemia #CERTCells

Discover how Dr. Jackie Cook and Dr. Frances Elmslie are pushing to streamline genomics adoption in the NHS. The article covers the push for faster rare disease diagnosis, the need for greater clinical literacy, and the role of AI in this exciting field. https://bit.ly/4bxhPQZ

In this week's 'In the Thick of It,' Kasey Woleben from Cure Mito Foundation discusses accelerating research for Leigh syndrome. Discover their top asks of industry, including the urgent need for paediatric clinical trials and responsible data sharing. https://bit.ly/4uBV7jo

Discover how collaboration, patient-centric design, and better data interoperability are accelerating rare disease research. From public-private partnerships to regulatory reform, learn the key initiatives driving progress and ensuring the patient voice is heard. https://bit.ly/4bqdI8Z

We asked our community "Do you have a clear communication plan for study/trial awareness and recruitment?".  Every week insights brings you the latest views from your global RARE community.  https://bit.ly/4u8P5qd

Joseph Burt, MHRA, spoke at the recent Westminster Health Forum Policy Conference on genomics in the UK #MHRA #Genomics #UKRegulation #AIDiagnostics #PrecisionMedicine https://bit.ly/4uenjIO#MHRA

Explore the future of UK genomics, from therapeutic advances for rare diseases to crucial policy debates on genomic data sharing. Experts discuss the need for innovation and a legal ban on the use of genomic data by insurers to prevent discrimination. https://bit.ly/4csczQA

Rare Disease Day 2026 has concluded, but the journey continues. Our latest article from our editor-in-chief, Nicola, acknowledges the emotional aftermath and calls for continued support to combat advocacy fatigue. https://bit.ly/46KznY7

The lack of research visibility in treatment journeys leaves patients unprepared for clinical trial participation. Learn how acknowledging the emotional and practical realities, especially in rare disease, can build trust and improve engagement. https://bit.ly/4rNcdZA

Genomics enters NHS labs by 2035 for routine, preventative care! Dr Ellen Thomas shares how this speeds diagnoses and bespoke treatments for kids with cancer and rare genetic disorders. https://bit.ly/4qWPhWA

The era of broad marketing for rare diseases is over. PharmaForceIQ's Hemal Somaiya shows how data-driven, hyper-personalised media engages HCPs, speeding patient access and cutting waste. https://bit.ly/4b0KYno

Don't miss Part Two! We explore how policy, MHRA's new pathways, reimbursement reform, and patient voices drive rare disease treatment access in the UK. https://bit.ly/4kTudie

In this week's "in the thick of it," Carrie Fulcher, Chair of Hope for Hypothalamic Hamartomas UK, highlights the top three unmet needs for the HH community: targeted seizure treatment, improved surgical options, and more precise imaging. https://bit.ly/40uJRXU

Read our exclusive interview with Dr. Nadine Großmann, a rare disease researcher and FOP expert, as she discusses her career path, the opportunities and barriers for women in her field, and her cutting-edge PhD work on age-dependent disease progression. https://bit.ly/4qWe0uc