RARE Revolution insider®
@rarerevinsider.bsky.social
Staying ahead in the world of rare disease isn’t a luxury…it’s a necessity. RARE Revolution insider® is a life sciences and rare disease publication for professionals, bridging the divide between industry and the community you serve.
We asked our community "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?". insights brings you the latest views from your global RARE community. https://bit.ly/4qf6jPT
December 19, 2025 at 12:00 PM
We asked our community "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?". insights brings you the latest views from your global RARE community. https://bit.ly/4qf6jPT
Our new poll reveals glaring gaps in how patient advocacy organisations work with the pharma industry. Key issues include overly complex MSAs, the need for unrestricted funding, and a critical lack of communication and feedback on co-design projects.
https://bit.ly/44tIjjt
https://bit.ly/44tIjjt
December 18, 2025 at 12:00 PM
Our new poll reveals glaring gaps in how patient advocacy organisations work with the pharma industry. Key issues include overly complex MSAs, the need for unrestricted funding, and a critical lack of communication and feedback on co-design projects.
https://bit.ly/44tIjjt
https://bit.ly/44tIjjt
Discover how the 'Macau Fast-Track' is revolutionising access to paediatric orphan drugs in Greater China. By leveraging existing approvals, manufacturers can deliver therapies to children in months, not years. https://bit.ly/48YrNcF
December 17, 2025 at 12:00 PM
Discover how the 'Macau Fast-Track' is revolutionising access to paediatric orphan drugs in Greater China. By leveraging existing approvals, manufacturers can deliver therapies to children in months, not years. https://bit.ly/48YrNcF
Following selection for the Morgan Stanley Inclusive and Sustainable Ventures Lab, Kate Cameron's team is poised to deliver human-relevant models that can replace animal testing.
https://rarerevolutionmagazine.com/rare-insider/cytochroma-making-a-splash-with-morgan-stanley-at-time-square/
https://rarerevolutionmagazine.com/rare-insider/cytochroma-making-a-splash-with-morgan-stanley-at-time-square/
December 16, 2025 at 12:01 PM
Following selection for the Morgan Stanley Inclusive and Sustainable Ventures Lab, Kate Cameron's team is poised to deliver human-relevant models that can replace animal testing.
https://rarerevolutionmagazine.com/rare-insider/cytochroma-making-a-splash-with-morgan-stanley-at-time-square/
https://rarerevolutionmagazine.com/rare-insider/cytochroma-making-a-splash-with-morgan-stanley-at-time-square/
Meet Patricia Weltin, CEO/Founder of Beyond the Diagnosis, and learn about her advocacy journey, the internationally recognised art exhibit, and her exciting new school programme, Beyond the Lesson.
https://rarerevolutionmagazine.com/rare-insider/patricia-weltin-of-beyond-the-diagnosis/
https://rarerevolutionmagazine.com/rare-insider/patricia-weltin-of-beyond-the-diagnosis/
December 15, 2025 at 12:01 PM
Meet Patricia Weltin, CEO/Founder of Beyond the Diagnosis, and learn about her advocacy journey, the internationally recognised art exhibit, and her exciting new school programme, Beyond the Lesson.
https://rarerevolutionmagazine.com/rare-insider/patricia-weltin-of-beyond-the-diagnosis/
https://rarerevolutionmagazine.com/rare-insider/patricia-weltin-of-beyond-the-diagnosis/
We asked our community "Were you clear on the motivation for collecting the data on your quality of life?". insights brings you the latest views from your global RARE community. To access insights click below.
https://bit.ly/4pbnFwO
https://bit.ly/4pbnFwO
December 12, 2025 at 12:00 PM
We asked our community "Were you clear on the motivation for collecting the data on your quality of life?". insights brings you the latest views from your global RARE community. To access insights click below.
https://bit.ly/4pbnFwO
https://bit.ly/4pbnFwO
Clinical trials for rare diseases face unique challenges, from limited patient pools to high costs. With 85% of trials failing to recruit on time, it's clear that patient experience must be prioritised.
https://rarerevolutionmagazine.com/rare-insider/understanding-the-true-cost-of-clinical-trials/
https://rarerevolutionmagazine.com/rare-insider/understanding-the-true-cost-of-clinical-trials/
Understanding the true cost of clinical trials
Alicia Staly, chief patient officer for Medidata, discusses the true cost of clinical trails for rare diseases
rarerevolutionmagazine.com
December 11, 2025 at 12:00 PM
Clinical trials for rare diseases face unique challenges, from limited patient pools to high costs. With 85% of trials failing to recruit on time, it's clear that patient experience must be prioritised.
https://rarerevolutionmagazine.com/rare-insider/understanding-the-true-cost-of-clinical-trials/
https://rarerevolutionmagazine.com/rare-insider/understanding-the-true-cost-of-clinical-trials/
Discover how medical affairs is bridging the gap between scientific innovation and equitable patient access in rare diseases.
https://rarerevolutionmagazine.com/rare-insider/bridging-innovation-and-access-how-medical-affairs-shapes-the-future-of-rare-disease-therapies/
https://rarerevolutionmagazine.com/rare-insider/bridging-innovation-and-access-how-medical-affairs-shapes-the-future-of-rare-disease-therapies/
December 10, 2025 at 12:00 PM
Discover how medical affairs is bridging the gap between scientific innovation and equitable patient access in rare diseases.
https://rarerevolutionmagazine.com/rare-insider/bridging-innovation-and-access-how-medical-affairs-shapes-the-future-of-rare-disease-therapies/
https://rarerevolutionmagazine.com/rare-insider/bridging-innovation-and-access-how-medical-affairs-shapes-the-future-of-rare-disease-therapies/
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 across the globe
https://bit.ly/4pLIS0g
https://bit.ly/4pLIS0g
December 9, 2025 at 12:00 PM
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 across the globe
https://bit.ly/4pLIS0g
https://bit.ly/4pLIS0g
Is the path to scientific genius the same for everyone? Despite progress in headcounts, gender inequality still impacts women in science through bias in employment, recognition, and funding.
https://bit.ly/4izhctd
https://bit.ly/4izhctd
December 8, 2025 at 12:00 PM
Is the path to scientific genius the same for everyone? Despite progress in headcounts, gender inequality still impacts women in science through bias in employment, recognition, and funding.
https://bit.ly/4izhctd
https://bit.ly/4izhctd
BridgeBio's FORTIFY phase 3 study is a turning point for limb-girdle muscular dystrophy (LGMD) patients. Chief Medical Officer Dr. Doug Sproule shares the promising results.
https://rarerevolutionmagazine.com/rare-insider/doug-sproule-fortify-studyunpicking-the-top-line-analysis
https://rarerevolutionmagazine.com/rare-insider/doug-sproule-fortify-studyunpicking-the-top-line-analysis
December 5, 2025 at 12:00 PM
BridgeBio's FORTIFY phase 3 study is a turning point for limb-girdle muscular dystrophy (LGMD) patients. Chief Medical Officer Dr. Doug Sproule shares the promising results.
https://rarerevolutionmagazine.com/rare-insider/doug-sproule-fortify-studyunpicking-the-top-line-analysis
https://rarerevolutionmagazine.com/rare-insider/doug-sproule-fortify-studyunpicking-the-top-line-analysis
We asked our community "Do you feel the quality-of-life assessment form adequately allowed you to convey the full impact of your condition, or that of your loved one, and what a good quality of life means to you, or your loved one?". Read the results here: https://bit.ly/49L6WvC
December 4, 2025 at 12:00 PM
We asked our community "Do you feel the quality-of-life assessment form adequately allowed you to convey the full impact of your condition, or that of your loved one, and what a good quality of life means to you, or your loved one?". Read the results here: https://bit.ly/49L6WvC
Grab a sneak peak of our interview with Doug Sproule, chief medical officer, ML solutions, a BridgeBio company, on FORTIFY, a phase 3 study for limb-girdle muscular dystrophy.
Watch here: https://rarerevolutionmagazine.com/rare-insider/doug-sproule-fortify-studyunpicking-the-top-line-analysis
Watch here: https://rarerevolutionmagazine.com/rare-insider/doug-sproule-fortify-studyunpicking-the-top-line-analysis
December 3, 2025 at 12:00 PM
Grab a sneak peak of our interview with Doug Sproule, chief medical officer, ML solutions, a BridgeBio company, on FORTIFY, a phase 3 study for limb-girdle muscular dystrophy.
Watch here: https://rarerevolutionmagazine.com/rare-insider/doug-sproule-fortify-studyunpicking-the-top-line-analysis
Watch here: https://rarerevolutionmagazine.com/rare-insider/doug-sproule-fortify-studyunpicking-the-top-line-analysis
The future of patient advocacy needs new leaders. With many groups facing aging leadership, pharma has a pivotal role to play. Michael Wilbur explains how industry can meaningfully invest in the next generation
https://rarerevolutionmagazine.com/rare-insider/rethinking-youth-engagement/
https://rarerevolutionmagazine.com/rare-insider/rethinking-youth-engagement/
December 2, 2025 at 12:00 PM
The future of patient advocacy needs new leaders. With many groups facing aging leadership, pharma has a pivotal role to play. Michael Wilbur explains how industry can meaningfully invest in the next generation
https://rarerevolutionmagazine.com/rare-insider/rethinking-youth-engagement/
https://rarerevolutionmagazine.com/rare-insider/rethinking-youth-engagement/
In this powerful 'Women in RARE' feature, meet Martine Pergent, President of IPOPI, who shares her inspiring journey from personal support to global Primary Immunodeficiency (PID) advocacy.
https://rarerevolutionmagazine.com/rare-insider/martine-pergent-of-ipopi/
https://rarerevolutionmagazine.com/rare-insider/martine-pergent-of-ipopi/
December 1, 2025 at 12:00 PM
In this powerful 'Women in RARE' feature, meet Martine Pergent, President of IPOPI, who shares her inspiring journey from personal support to global Primary Immunodeficiency (PID) advocacy.
https://rarerevolutionmagazine.com/rare-insider/martine-pergent-of-ipopi/
https://rarerevolutionmagazine.com/rare-insider/martine-pergent-of-ipopi/
We asked our community "Have you ever completed a quality-of-life assessment form?". insights brings you the latest views from your global RARE community. To access insights click below. https://rarerevolutionmagazine.com/rare-insider/have-you-ever-completed-a-quality-of-life-assessment-form/
November 28, 2025 at 12:00 PM
We asked our community "Have you ever completed a quality-of-life assessment form?". insights brings you the latest views from your global RARE community. To access insights click below. https://rarerevolutionmagazine.com/rare-insider/have-you-ever-completed-a-quality-of-life-assessment-form/
Breakthroughs in rare disease research are bringing personalised treatments into sight. Read our interview with Dr Michael Mannstadt, principal investigator of the landmark CALIBRATE clinical trial
https://rarerevolutionmagazine.com/rare-insider/personalised-treatments-in-sight-for-adh1/
https://rarerevolutionmagazine.com/rare-insider/personalised-treatments-in-sight-for-adh1/
November 27, 2025 at 12:00 PM
Breakthroughs in rare disease research are bringing personalised treatments into sight. Read our interview with Dr Michael Mannstadt, principal investigator of the landmark CALIBRATE clinical trial
https://rarerevolutionmagazine.com/rare-insider/personalised-treatments-in-sight-for-adh1/
https://rarerevolutionmagazine.com/rare-insider/personalised-treatments-in-sight-for-adh1/
🚨 Happening today! RARE REV-inar episode 023 - Strengthening coordination between the health system and industry to improve patient access kicks off at 12pm GMT.
It’s not too late—sign up free here: https://bit.ly/RR-UCB-Webinar
It’s not too late—sign up free here: https://bit.ly/RR-UCB-Webinar
November 27, 2025 at 10:00 AM
🚨 Happening today! RARE REV-inar episode 023 - Strengthening coordination between the health system and industry to improve patient access kicks off at 12pm GMT.
It’s not too late—sign up free here: https://bit.ly/RR-UCB-Webinar
It’s not too late—sign up free here: https://bit.ly/RR-UCB-Webinar
Open Label Extension (OLE) studies are vital in clinical research, particularly for chronic and rare conditions like Developmental and Epileptic Encephalopathies (DEEs)
https://rarerevolutionmagazine.com/rare-insider/understanding-open-label-extensions-in-clinical-trials-purpose-design-and-benefit/
https://rarerevolutionmagazine.com/rare-insider/understanding-open-label-extensions-in-clinical-trials-purpose-design-and-benefit/
November 26, 2025 at 12:00 PM
Open Label Extension (OLE) studies are vital in clinical research, particularly for chronic and rare conditions like Developmental and Epileptic Encephalopathies (DEEs)
https://rarerevolutionmagazine.com/rare-insider/understanding-open-label-extensions-in-clinical-trials-purpose-design-and-benefit/
https://rarerevolutionmagazine.com/rare-insider/understanding-open-label-extensions-in-clinical-trials-purpose-design-and-benefit/
In the thick of it: Putting you in the heart of the rare community with the SMS Foundation UK. This week, Executive Director Leeann Stevenson outlines the top three unmet needs for Smith-Magenis syndrome
https://bit.ly/3XDIq8a
https://bit.ly/3XDIq8a
November 25, 2025 at 12:00 PM
In the thick of it: Putting you in the heart of the rare community with the SMS Foundation UK. This week, Executive Director Leeann Stevenson outlines the top three unmet needs for Smith-Magenis syndrome
https://bit.ly/3XDIq8a
https://bit.ly/3XDIq8a
Grab a sneak peak of our interview with Dr Michael Mannstadt, principal investigator of the landmark CALIBRATE clinical trial, as he discusses the rare genetic disorder ADH1
https://rarerevolutionmagazine.com/rare-insider/dr-michael-mannstadt-next-steps-for-the-calibrate-clinical-trial-for-adh1/
https://rarerevolutionmagazine.com/rare-insider/dr-michael-mannstadt-next-steps-for-the-calibrate-clinical-trial-for-adh1/
November 24, 2025 at 12:00 PM
Grab a sneak peak of our interview with Dr Michael Mannstadt, principal investigator of the landmark CALIBRATE clinical trial, as he discusses the rare genetic disorder ADH1
https://rarerevolutionmagazine.com/rare-insider/dr-michael-mannstadt-next-steps-for-the-calibrate-clinical-trial-for-adh1/
https://rarerevolutionmagazine.com/rare-insider/dr-michael-mannstadt-next-steps-for-the-calibrate-clinical-trial-for-adh1/
Hear from Nico Reynders, General Manager at UCB UK & Ireland and Vice Chair of the European Medicines Group. Nico champions patient-focused solutions and policy change for rare diseases.
Sign up free: https://bit.ly/RR-UCB-Webinar
#Pharma #PolicyChange #RareDisease #RARERevinar
Sign up free: https://bit.ly/RR-UCB-Webinar
#Pharma #PolicyChange #RareDisease #RARERevinar
November 24, 2025 at 9:00 AM
Hear from Nico Reynders, General Manager at UCB UK & Ireland and Vice Chair of the European Medicines Group. Nico champions patient-focused solutions and policy change for rare diseases.
Sign up free: https://bit.ly/RR-UCB-Webinar
#Pharma #PolicyChange #RareDisease #RARERevinar
Sign up free: https://bit.ly/RR-UCB-Webinar
#Pharma #PolicyChange #RareDisease #RARERevinar
Dr Charlotte Roy from Muscular Dystrophy UK is joining our panel! With a background in molecular biology and advocacy, Charlotte brings both expertise and empathy to the rare disease conversation.
Register: https://bit.ly/RR-UCB-Webinar
#MuscularDystrophy #PatientAdvocacy #RareDisease #RARERevinar
Register: https://bit.ly/RR-UCB-Webinar
#MuscularDystrophy #PatientAdvocacy #RareDisease #RARERevinar
November 21, 2025 at 2:00 PM
Dr Charlotte Roy from Muscular Dystrophy UK is joining our panel! With a background in molecular biology and advocacy, Charlotte brings both expertise and empathy to the rare disease conversation.
Register: https://bit.ly/RR-UCB-Webinar
#MuscularDystrophy #PatientAdvocacy #RareDisease #RARERevinar
Register: https://bit.ly/RR-UCB-Webinar
#MuscularDystrophy #PatientAdvocacy #RareDisease #RARERevinar
Grab a sneak peak of our interview with Dr Amélie Lothe, medical community head for rare epilepsies, UCB where she discusses the rationale, design, ethical considerations and impact of open label extensions
https://bit.ly/49UB5sa
https://bit.ly/49UB5sa
November 21, 2025 at 1:00 PM
Grab a sneak peak of our interview with Dr Amélie Lothe, medical community head for rare epilepsies, UCB where she discusses the rationale, design, ethical considerations and impact of open label extensions
https://bit.ly/49UB5sa
https://bit.ly/49UB5sa
We asked our community "What does a good quality of life look like to you?". insights brings you the latest views from your global RARE community. To access insights click below.
https://rarerevolutionmagazine.com/rare-insider/what-does-a-good-quality-of-life-look-like-to-you
https://rarerevolutionmagazine.com/rare-insider/what-does-a-good-quality-of-life-look-like-to-you
November 20, 2025 at 1:01 PM
We asked our community "What does a good quality of life look like to you?". insights brings you the latest views from your global RARE community. To access insights click below.
https://rarerevolutionmagazine.com/rare-insider/what-does-a-good-quality-of-life-look-like-to-you
https://rarerevolutionmagazine.com/rare-insider/what-does-a-good-quality-of-life-look-like-to-you