Highlights:

Genetic Fix or False Hope? The Reality of Gene Therapy in Rare Conditions

FDA Approves First Gene Therapy for Wiskott–Aldrich Syndrome, Milestone for Non-Profit Research

The approval of Waskyra for Wiskott–Aldrich syndrome demonstrates that gene therapies can successfully reach regulatory approval when developed by non-profit organizations. This milestone may accelerate future therapeutic innovation, broaden investment in rare disease research, and expand access to curative-intent treatments for underserved patient populations.

Dec 19, 20252 min read

Man smiling in a doctor's office, wearing a blue shirt. Medical equipment and an exam bed are in the background. Warm, relaxed atmosphere.

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 26, 20257 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 23, 20255 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 21, 20257 min read

Living Rare Editorials

Woman in a wheelchair on cracked desert ground, looking contemplative. She's wearing a blue shirt and beige pants under a hazy sky.

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 29, 202512 min read

DNA strand and virus particle in a lab, with a spark indicating interaction; blue and orange colors dominate the scene.

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 11, 20258 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 28, 20254 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 26, 20255 min read

Patient Advocacy & Community Activism

Tulsi plant beside a glass jar of powder and a wooden spoon on a table, with a cup of tea in the background.

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 2, 20254 min read

Man facing a maze symbolizing "Prior Authorization" with a sign saying "Rare Disease Care" behind him. The scene suggests complexity and bureaucratic challenges.

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 24, 20259 min read

Young boy in a navy shirt sits on a black wheelchair in a room with beige walls and light curtains, appearing contemplative while living with Spinal Muscular Atrophy.

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 17, 20259 min read

Two concerned adults and a child hold a $28,000 medical bill at a table, showing the economics of Rare Diseases. They're indoors with soft lighting.

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 2, 20258 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 27, 20255 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 22, 20255 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