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A Historic Breakthrough in Huntington’s Disease: Gene Therapy Offers New Hope
For decades, Huntington’s disease had no treatment that could alter its course. The new AMT 130 gene therapy trial shows promise in slowing disease progression, marking a historic milestone for patients, families, and the broader rare disease community.
Sep 267 min read
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
Rare Disease Families Are on the Frontlines of Climate Change
This article explores how climate change is deepening the crisis for rare disease patients worldwide, and why the path forward must include policies and protections tailored to this uniquely at-risk population.
Aug 2912 min read
Genetic Fix or False Hope? The Reality of Gene Therapy in Rare Conditions
Explore the reality of Gene Therapy in rare conditions. Is it a genetic fix or false hope?
Aug 118 min read
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
How Holy Basil (Tulsi) Lowers Cortisol and Why It Matters for Rare Disease Patients
Holy basil (Ocimum sanctum), or tulsi, is more than a sacred herb in Ayurveda—it’s an adaptogen with proven benefits for stress relief. Research indicates that tulsi can help lower cortisol levels, enhance sleep quality, and promote overall well-being.
Oct 24 min read
Insurance Denials in Rare Disease Care: Systemic Flaws in Coverage
Insurance denials create devastating barriers for rare disease patients, delaying essential treatments and driving up costs. This article explores systemic flaws in coverage policies, the appeal process, and new reforms aiming to improve equity and access to rare disease care.
Sep 249 min read
A Closer Look at Spinal Muscular Atrophy: Symptoms, Types, and Management
Spinal Muscular Atrophy (SMA) is a rare genetic disorder that affects the motor neurons, leading to muscle weakness and atrophy.
Sep 179 min read
The Economics of Rare Diseases: How Financial Burdens Impact Patients and their Families
Rare diseases don’t just affect health—they place overwhelming financial and emotional strain on patients and caregivers. From costly treatments to lost wages, this article uncovers the hidden burden of rare diseases and why systemic policy change is critical.
Sep 28 min read
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
Thought Leadership in Mental Health For Rare Diseases
A discussion with Mental Health for Rare Thought Leader JC Muyl.
May 7, 20247 min read
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