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.

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.

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.

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.

Spinal Muscular Atrophy (SMA) is a rare genetic disorder that affects the motor neurons, leading to muscle weakness and atrophy.

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.

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.

Digital biomarkers are unlocking earlier detection, real-time monitoring, and personalized interventions for rare disease patients, reshaping how we diagnose and manage these complex conditions.

Explore the reality of Gene Therapy in rare conditions. Is it a genetic fix or false hope?

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.

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.

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.

Explore Montana's Medical Experimentation law. Is it a groundbreaking step or a risky gamble?

In a groundbreaking first, doctors developed a custom gene- editing therapy in just seven months to treat a baby with a deadly and ultra-rare metabolic disorder.

Discover the complexities of CHOPS syndrome, a rare genetic disorder impacting multiple body systems.

Join us this Rare Disease Day to advocate for an inclusive healthcare system.

The TikTok ban in the US has far-reaching consequences, especially for the rare disease community.

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

In a controversial move, the FDA has quietly removed its draft guidance on clinical trial diversity, sparking concerns over inclusivity.

Cushing’s Syndrome is a rare but serious endocrine disorder caused by prolonged exposure to high levels of cortisol.

Idiopathic Ketotic Hypoglycemia (IKH) is a rare metabolic disorder often overlooked in children.

Heather F., co-founder of Level 5 Insights, is redefining healthcare market research with a mission rooted in empathy, inclusion, & advocacy

Pharma companies must respect the privacy of rare disease communities on social media.

Explore how Rare360 empowers the rare disease community through advocacy and trust-building.