Author | Lightnews

RARE Revolution insider® @rarerevinsider.bsky.social · 58m

"When it comes to global awareness campaigns, local context is key! This insightful article from Michael Wilbur on 'The Problem with the Zebra' highlights why campaigns, especially for Rare Disease Day, must be rooted in local leadership.
https://bit.ly/4qfyy1L

The problem with the zebra: Why global awareness campaigns must be rooted in local leadership

Michael Wilbur discusses the problem with the zebra and why global awareness campaigns must be rooted in local leadership.

rarerevolutionmagazine.com

RARE Revolution insider® @rarerevinsider.bsky.social · 1d

We asked our community "If you/your advocacy organisation have participated in any co-design activities with industry, did you feel the outcomes reflected your community’s input to your satisfaction?". insights brings you the latest views from your global RARE community.
https://bit.ly/4oO29xN

RARE Revolution insider® @rarerevinsider.bsky.social · 2d

A recent RARE Revolution poll reveals that diagnostic pathways and service provision for rare disease patients are falling short. The findings highlight the urgent need for system-wide reform.
https://bit.ly/3Li4RwG

RARE Revolution poll finds diagnostic pathways and service provision is failing patients

RARE Revolution finds a mixed diagnostic and service provision picture showing a pattern of delayed diagnoses and services falling short.

rarerevolutionmagazine.com

RARE Revolution insider® @rarerevinsider.bsky.social · 3d

In this insightful feature, early-career professionals in the life sciences share candid reflections on navigating the rare disease space.

https://rarerevolutionmagazine.com/rare-insider/finding-your-feet-in-the-field-lessons-from-those-in-the-life-sciences/

Finding your feet in the field: Lessons from those in the life sciences

It can be hard to find a job you want, but many are in a similar position. Your dream job doesn't have to be fixed, life can take a new path.

rarerevolutionmagazine.com

1

RARE Revolution insider® @rarerevinsider.bsky.social · 4d

Holly Landrum Peay, Senior Director at Faegre Drinker Consulting, shares her inspiring journey from genetic counselling to leading patient-centred research in rare disease

https://rarerevolutionmagazine.com/rare-insider/holly-landrum-peay-of-faegre-drinker-consulting/

Holly Landrum Peay of Faegre Drinker Consulting

Holly Landrum Peay, "I was motivated to my current career path by a long-standing awareness that the ‘end user,’ must be the central focus."

rarerevolutionmagazine.com

RARE Revolution insider® @rarerevinsider.bsky.social · 7d

In the highly regulated world of clinical research, an innovative approach is emerging that promises to transform how treatments are developed. One2Treat, a pioneering company, is challenging traditional clinical trial design by putting patients at the heart of the process.
https://bit.ly/One2Treat

Revolutionising clinical trials: One2Treat’s patient-centric approach to drug development

One2Treat is pioneering a paradigm shift in how medical treatments are conceptualised, developed and evaluated.

bit.ly

RARE Revolution insider® @rarerevinsider.bsky.social · 8d

RARE Revolution poll reveals patient needs are worryingly overlooked in clinical trial design. insights brings you the latest views from your global RARE community.
https://rarerevolutionmagazine.com/rare-insider/rare-revolution-poll-reveals-patient-needs-are-worryingly-overlooked-in-clinical-trials

RARE Revolution insider® @rarerevinsider.bsky.social · 9d

Bob Stevens, CEO of the MPS Society, argues that traditional randomised controlled trials are unethical for rare and ultra-rare diseases, especially when neurological involvement leads to irreversible decline. https://bit.ly/BobRCTs

RARE Revolution insider® @rarerevinsider.bsky.social · 10d

Discover the vital work of Action for XP, the UK's sole charity supporting individuals and families affected by xeroderma pigmentosum (XP). This rare genetic condition causes extreme UV sensitivity, leading to life-threatening cancers. https://bit.ly/A4XP

RARE Revolution insider® @rarerevinsider.bsky.social · 11d

In this insightful article, Owen Marks explores the crucial stepping stones to successful rare disease medicine launches in the UK. He highlights the "Rare Disease Gap" in funding, the imperative for early engagement with communities.
https://bit.ly/RareSteppingStones

1

RARE Revolution insider® @rarerevinsider.bsky.social · 14d

When was the last time you read something that mattered?

RARE Revolution insider® brings you straight to the people that matter.

Trusted stories. Expert perspectives. No spin.

Claim your free October trial: https://my.rarerevolutionmagazine.com/

RARE Revolution insider® @rarerevinsider.bsky.social · 14d

Discover how the UK's current medicines pricing and access policies are failing those with rare conditions. Henry Burkitt, MD at Oxygen Strategy, calls for a complete rethink.
https://rarerevolutionmagazine.com/rare-insider/when-policy-fails-people-reimagining-medicines-access-for-rare-conditions/

RARE Revolution insider® @rarerevinsider.bsky.social · 15d

We asked our community "If you/your advocacy organisation have participated in any co-design activities with industry were the activity/project outcomes fed back to you?". insights brings you the latest views from your global RARE community.
https://bit.ly/CoDesignInsights

If you/your advocacy organisation have participated in any co-design activities with industry were the activity/project outcomes fed back to you?

If you/your advocacy organisation have participated in any co-design activities with industry were the outcomes fed back to you?

rarerevolutionmagazine.com

RARE Revolution insider® @rarerevinsider.bsky.social · 16d

Innovative trial designs are reshaping rare disease drug development, offering smarter, faster, and more inclusive approaches. Dr Billy Amzal of Phastar spotlights key tools. https://rarerevolutionmagazine.com/rare-insider/how-innovative-trial-designs-de-risk-drug-development-in-rare-diseases/

How innovative trial designs de-risk drug development in rare diseases

Dr Billy Amzal, leading strategic consulting at Phastar, explores the current innovations transforming drug development for rare disease.

rarerevolutionmagazine.com

RARE Revolution insider® @rarerevinsider.bsky.social · 17d

Stacy Pike-Langenfeld, co-founder of KrabbeConnect, shares her powerful journey from grief to purpose, championing patient-led research and data-driven advocacy in rare disease. https://rarerevolutionmagazine.com/rare-insider/stacy-pike-langenfeld-of-krabbeconnet/

Stacy Pike-Langenfeld of KrabbeConnect

Stacy Pike Langenfeld, "I want families affected by rare disease to have trusted information and to help amplify the lived experience."

rarerevolutionmagazine.com

RARE Revolution insider® @rarerevinsider.bsky.social · 17d

Time is short. Complexity is high.
RARE Revolution insider® brings you straight to the strategies that matter and highlights the people, innovations, and ideas shaping rare disease development. Independent. Intelligent. Accessible.
Free trial this October: https://my.rarerevolutionmagazine.com/

1

RARE Revolution insider® @rarerevinsider.bsky.social · 18d

Dr Andrés Moreno-Estrada and Carmina Barberena-Jonas from Cinvestav unveil MexVar, a new research platform revealing specific risk variants. Early findings could transform treatment. https://rarerevolutionmagazine.com/rare-insider/mexvar-helping-to-increase-the-scientific-independence-of-mexico/

MexVar: Helping to increase the scientific independence of Mexico

Andrés Moreno-Estrada and Carmina Barberena-Jonas, Cinvestav, discuss their new research platform, building scientific independence in LATAM.

rarerevolutionmagazine.com

RARE Revolution insider® @rarerevinsider.bsky.social · 20d

Don’t just search. Find what matters. RARE Revolution insider® subscribers get full access to insights powered by our librarian Cognito.

Every search is across trusted, compliant, approved content.

Fast, accurate, independent.

Free 1-month trial here: https://my.rarerevolutionmagazine.com/

RARE Revolution insider® @rarerevinsider.bsky.social · 21d

Discover how Sumair Nizamuddin from Nuffield Bio is tackling mistrust in healthcare by addressing medical racism and advocating for community engagement. Learn more! https://rarerevolutionmagazine.com/rare-insider/breaking-the-cycle-rebuilding-trust-in-medical-research-and-delivery/

Breaking the cycle: rebuilding trust in medical research and delivery

Sumair Nizamuddin shares how to address issues of mistrust of healthcare processes, we need to delve further back in history.

rarerevolutionmagazine.com

RARE Revolution insider® @rarerevinsider.bsky.social · 22d

Have you participated in co-design with industry? Access insights from our global RARE community: https://rarerevolutionmagazine.com/rare-insider/have-you-or-your-advocacy-organisation-taken-part-in-any-co-design-activities-or-projects-with-industry/

RARE Revolution insider® @rarerevinsider.bsky.social · 22d

Too much noise. Not enough clarity. RARE Revolution insider® gets straight to what matters.
Independent stories, human voices, new ways of doing things in rare disease. Not PR. Not hype. Just clarity. Try your free month in October.

https://my.rarerevolutionmagazine.com/

RARE Revolution insider® @rarerevinsider.bsky.social · 23d

In "The Power of Words," Florence Cornish illustrates how clear science language benefits patients, professionals, and policymakers. Get impactful writing tips! https://rarerevolutionmagazine.com/rare-insider/the-power-of-words-how-should-we-write-about-science #PlainLanguage #MedicalWriting

RARE Revolution insider® @rarerevinsider.bsky.social · 24d

Join us this week to explore the vital work of Childhood Tumour Trust with CEO Vanessa Martin! Learn about #NF1 and its unmet needs. https://rarerevolutionmagazine.com/rare-insider/putting-you-in-the-heart-of-the-rare-community-childhood-tumour-trust/ #RareDisease #PatientAdvocacy

1

RARE Revolution insider® @rarerevinsider.bsky.social · 25d

Revolutionising rare disease pharmacovigilance! AI is transforming safety monitoring for diverse patient populations.
https://rarerevolutionmagazine.com/rare-insider/strengthening-rare-disease-pharmacovigilance-with-curated-real-world-data/
#Pharmacovigilance #RealWorldData #RWD #AIinHealthcare

RARE Revolution insider® @rarerevinsider.bsky.social · Sep 22

Discover how Prof. Gareth Baynam's 'Lyfe Languages' project bridges healthcare and Indigenous culture by translating medical terms! #IndigenousHealth #HealthcareInnovation #LyfeLanguages #RareDisease rarerevolutionmagazine.com/rare-insider...

From songlines to science: Bridging healthcare and Indigenous culture

Gareth Baynam has pioneered an innovative project translating medical terminology into Aboriginal languages.

rarerevolutionmagazine.com

1 2