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News Briefs
Tragic Updates from Sarepta’s Gene Therapy Programs
Tragic deaths in Sarepta’s gene therapy trials have deeply impacted the rare disease community, raising concerns about liver risks, FDA responses, and treatment access.
Jul 235 min read
Inside the Orphan Drug Act: Incentives Fueling Rare Disease Therapies
This article breaks down what the ODA is, why it matters, the challenges that remain, and what patients and caregivers need to know to advocate for continued progress in rare disease care.
Jul 217 min read
Montana’s Medical Experimentation Law: A New Frontier—or a Dangerous Gamble?
Explore Montana's Medical Experimentation law. Is it a groundbreaking step or a risky gamble?
Jul 113 min read
Living Rare Editorials
Rare Disease Day: A Call to Action for a More Inclusive Healthcare System
Join us this Rare Disease Day to advocate for an inclusive healthcare system.
Feb 284 min read
Silencing Connections: The Impact of Social Media Bans on Rare Disease Communities
The TikTok ban in the US has far-reaching consequences, especially for the rare disease community.
Feb 265 min read
Love Beyond Romance: Guide to Embracing Self-Care and Connection on Valentine’s Day
For many, Valentine’s Day is all about romantic gestures—but for those with rare diseases, it’s a powerful reminder to celebrate self-love f
Feb 145 min read
Patient Advocacy & Community Activism
Managing Dialysis When You Have a Rare Kidney Disorder
Dialysis can be different for people living with rare kidney disorders. Learn what to expect, how to navigate challenges, and find hope for the future.
6 days ago6 min read
Inside the Orphan Drug Act: Incentives Fueling Rare Disease Therapies
This article breaks down what the ODA is, why it matters, the challenges that remain, and what patients and caregivers need to know to advocate for continued progress in rare disease care.
Jul 217 min read
CHOPS Syndrome: Discovering the Complexities of a Rare Genetic Condition
Discover the complexities of CHOPS syndrome, a rare genetic disorder impacting multiple body systems.
Mar 36 min read
Thought Leaders
Redefining Market Research with Empathy and Inclusion: Insights from Heather Flaherty
Heather F., co-founder of Level 5 Insights, is redefining healthcare market research with a mission rooted in empathy, inclusion, & advocacy
Jan 275 min read
Repairing Trust and Supporting Community-based Advocacy: A Conversation with Rare360's Executive Director
Explore how Rare360 empowers the rare disease community through advocacy and trust-building.
Jan 225 min read
Championing Change: Brittany Jež on Bridging Gaps in Rare Disease Healthcare
From personal experience to professional impact, Brittany Jež is transforming rare disease diagnostics for the hEDS community and beyond
Dec 18, 20245 min read
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