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New IN this week

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How do new medicines get approved by the European Union?

By Florence Cornish, RARE Revolution
12 January 2026

How has the political landscape during 2025 impacted your company/the company you work for?

By Emma Bishop, RARE Revolution
12 January 2026

Flaminia Macchia, chief operating officer, Orphanet-AISBL

By CONTRIBUTOR
12 January 2026

Giacomo Chiesi: Forging a new path in genomic medicine with Arbor Biotechnologies

By Nicola Miller, RARE Revolution
12 January 2026

IN the know

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Why rare disease medicines can’t be priced like generics—and why that matters for patients

By Owen Marks, Omgen
12 January 2026

“Hope floats”

By Nicola Miller, RARE Revolution
5 January 2026

Rethinking youth engagement

By Michael Wilbur, MW Advocacy Solutions
1 December 2025

Newborn screening: when the same screen results in different outcomes—and why universality isn’t enough

By Amy Gaviglio, Connetics Consulting
17 November 2025

INnovation

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Cytochroma making a splash with Morgan Stanley at Time Square

By Nicola Miller, RARE Revolution
15 December 2025

What AI innovations like China’s DeepSeek could mean for rare diseases

By Julie Penfold, RARE Revolution
8 December 2025

“Spray and pray marketing no longer works”: finding the moments of opportunity

By Nicola Miller, RARE Revolution
10 November 2025

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

By CONTRIBUTOR
6 October 2025

IN motion

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Giacomo Chiesi: Forging a new path in genomic medicine with Arbor Biotechnologies

By Nicola Miller, RARE Revolution
12 January 2026

Steffen Thirstrup: medicine approval in Europe

By Emma Bishop, RARE Revolution
5 January 2026

Doug Sproule: FORTIFY Study—unpicking the top line analysis

By Nicola Miller, RARE Revolution
1 December 2025

Dr Michael Mannstadt: next steps for the CALIBRATE clinical trial for ADH1

By Nicola Miller, RARE Revolution
24 November 2025

Women IN RARE

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Flaminia Macchia, chief operating officer, Orphanet-AISBL

By CONTRIBUTOR
12 January 2026

Patricia Weltin of Beyond the Diagnosis

By CONTRIBUTOR
15 December 2025

Martine Pergent of IPOPI

By CONTRIBUTOR
1 December 2025

Serene Forte of Forte BioConsulting, LLC

By CONTRIBUTOR
17 November 2025

INcognito

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incognito: the secret life of a…rare disease board trustee

By CONTRIBUTOR
25 August 2025

incognito: the secret life of a…community CEO

By CONTRIBUTOR
11 August 2025

incognito: the secret life of an…advocacy group CEO

By CONTRIBUTOR
28 July 2025

incognito: the secret life of a…research CEO

By CONTRIBUTOR
14 July 2025

IN the thick of it

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Putting you in the heart of the rare community – SMS Foundation UK

By CONTRIBUTOR
24 November 2025

Putting you in the heart of the rare community – Action FCS

By CONTRIBUTOR
10 November 2025

Voices united: addressing the unmet needs of patient advocacy groups

By Emma Bishop, RARE Revolution
10 November 2025

Putting you in the heart of the rare community – Ring20 Research and Support UK CIO

By CONTRIBUTOR
27 October 2025

INsights

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How has the political landscape during 2025 impacted your company/the company you work for?

By Emma Bishop, RARE Revolution
12 January 2026

RARE Revolution poll finds glaring gaps when patient advocacy organisations work with industry

By Julie Penfold, RARE Revolution
15 December 2025

Have you, or your loved one, directly benefitted from a change in service, care or treatment options following the completion of a quality of life assessment form?

By Emma Bishop, RARE Revolution
15 December 2025

Were you clear on the motivation for collecting the data on your quality of life form?

By Emma Bishop, RARE Revolution
8 December 2025

IN person

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ISPOR Europe 2025, Tuesday 11th November—view from a newbie

By Rebecca Stewart, RARE Revolution
17 November 2025

The real conversations at World Orphan Drug Congress Europe

By Rebecca Stewart, RARE Revolution
5 November 2025

IN the loop

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How do new medicines get approved by the European Union?

By Florence Cornish, RARE Revolution
12 January 2026

Who gets to be a genius? The ongoing impact of gender bias in science

By Florence Cornish, RARE Revolution
8 December 2025

Bridging innovation and access: How medical affairs shapes the future of rare disease therapies

By CONTRIBUTOR
8 December 2025

Understanding open label extensions in clinical trials—purpose, design and benefit

By Nicola Miller, RARE Revolution
24 November 2025

IN the pipeline

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Turning point: FORTIFY phase 3 study for limb-girdle muscular dystrophy

By Nicola Miller, RARE Revolution
1 December 2025

Personalised treatments in sight for ADH1

By Nicola Miller, RARE Revolution
24 November 2025

Best-in-class anti-CD38 therapy for ITP

By Nicola Miller, RARE Revolution
8 September 2025

IN the round

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Macau first: a scalable entry route for paediatric orphan drugs into Greater China

By CONTRIBUTOR
15 December 2025

MexVar: Helping to increase the scientific independence of Mexico

By Julie Penfold, RARE Revolution
6 October 2025

From songlines to science: Bridging healthcare and Indigenous culture

By Emma Bishop, RARE Revolution
14 July 2025

Solving the unsolvable: a revolutionary new model for diagnosing rare diseases

By Emma Bishop, RARE Revolution
30 June 2025

IN the spotlight

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

By Nicola Miller, RARE Revolution
4 August 2025

AlphaRose Therapeutics on doing things differently

By Julie Penfold, RARE Revolution
23 June 2025
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