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RAREBite Newsletter Subscribers (Twice Weekly)
Magazine and RARE Round-Up Weekly Newsletter
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New IN this week
IN the know
INnovation
IN motion
Women IN RARE
INcognito
IN the thick of it
INsights
IN the spotlight
IN the round
IN the loop
IN the pipeline
IN person
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New IN this week
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Withdrawal of USAID is fanning winds of entrepreneurial change across Africa
By CONTRIBUTOR
23 February 2026
Rethinking consent: The unresolved challenge of genomic newborn screening
By Amy Gaviglio, Connetics Consulting
23 February 2026
Do you understand the difference between study/trial awareness and study/trial recruitment?
By Emma Bishop, RARE Revolution
23 February 2026
From ‘high conviction’ to patient impact: inside BIA’s UK Biotech Financing 2025 report – Part one
By Nicola Miller, RARE Revolution
23 February 2026
IN the know
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Rethinking consent: The unresolved challenge of genomic newborn screening
By Amy Gaviglio, Connetics Consulting
23 February 2026
Singing for your dinner
By Daniel Lewi, The CATS Foundation
16 February 2026
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
INnovation
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AI is creating a new Darwinian jobs market where the super employee is king
By CONTRIBUTOR
9 February 2026
Is it possible to predict and prevent seizures? The promise of a new algorithm
By Florence Cornish, RARE Revolution
2 February 2026
My daughter is going blind. We are building a new model for rare disease gene therapy
By CONTRIBUTOR
26 January 2026
Cytochroma making a splash with Morgan Stanley at Time Square
By Nicola Miller, RARE Revolution
15 December 2025
IN motion
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Dr Martin Turner and Rosie Lindup on BIA’s UK Biotech Financing 2025 report
By Nicola Miller, RARE Revolution
16 February 2026
Tim Szalay – striving to revolutionise biomedical research
By Nicola Miller, RARE Revolution
19 January 2026
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
Women IN RARE
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Lesa Brackbill, director of advocacy, Patient Advocacy Strategies
By CONTRIBUTOR
16 February 2026
Kris Pierce, strategic lead, Rare Disease NSW and founder, SCN2A Australia
By CONTRIBUTOR
9 February 2026
Fatima Scipione, vice president, Global Patient Affairs, Blueprint Medicines, a Sanofi company
By CONTRIBUTOR
26 January 2026
Flaminia Macchia, chief operating officer, Orphanet-AISBL
By CONTRIBUTOR
12 January 2026
INcognito
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incognito: the secret life of a…rare disease parent and charity founder
By CONTRIBUTOR
16 February 2026
incognito: the secret life of an…independent patient engagement consultant to industry
By CONTRIBUTOR
2 February 2026
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
IN the thick of it
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Putting you in the heart of the rare community – Timothy Syndrome Alliance (TSA)
By CONTRIBUTOR
9 February 2026
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
INsights
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Do you understand the difference between study/trial awareness and study/trial recruitment?
By Emma Bishop, RARE Revolution
23 February 2026
Who do you feel is responsible for study/trial awareness and recruitment?
By Emma Bishop, RARE Revolution
16 February 2026
How important is partnering with patient groups to the successes within your company?
By Emma Bishop, RARE Revolution
9 February 2026
What do you feel is the most pressing priority for successful rare disease drug discovery in 2026?
By Emma Bishop, RARE Revolution
2 February 2026
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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Accessing life-sustaining equipment: a silent systemic gap
By CONTRIBUTOR
23 February 2026
From ‘high conviction’ to patient impact: inside BIA’s UK Biotech Financing 2025 report – Part one
By Nicola Miller, RARE Revolution
23 February 2026
Key challenges in transition: how young people with rare diseases are falling through the net
By Becky Pender, RARE Revolution
2 February 2026
Curative ambitions: Chiesi Group and Arbor Biotechnologies forge a new path in genomic medicine
By Nicola Miller, RARE Revolution
19 January 2026
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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Withdrawal of USAID is fanning winds of entrepreneurial change across Africa
By CONTRIBUTOR
23 February 2026
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
IN the spotlight
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Programming the future of rare disease: ABYA Genomics’ AI revolution
By Nicola Miller, RARE Revolution
26 January 2026
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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