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Tag: Rare Disease

The Cost of Convenience - Why Administrative Hurdles Should Never Block Newborn Screening

The Cost of Convenience: Why Administrative Hurdles Should Never Block Newborn Screening

In policy meetings and committee rooms across the country, newborn screening conditions are regularly scored, debated, and weighed. Leaders evaluate analytical validity, treatment efficacy, and system capacity. But every so...

Published September 2, 2026
Why Newborn Screening Matters - The Difference Between a Diagnosis and a Future

Why Newborn Screening Matters – The Difference Between a Diagnosis and a Future

Learn why newborn screening is one of the most important public health tools for rare disease diagnosis, early intervention, health equity, and improved patient outcomes....
Published August 21, 2026
My Voice Matters - Living with Duchenne Muscular Dystrophy

My Voice Matters: Living with Duchenne Muscular Dystrophy  

Devin Argall shares his experience living with Duchenne muscular dystrophy and how small assumptions can impact identity, inclusion, and respect....
Published August 12, 2026
Recruitment Creates Awareness - Patient Stories Create Confidence

Recruitment Creates Awareness – Patient Stories Create Confidence

Learn how patient story videos build trust, improve engagement, and help sponsors create stronger clinical trial recruitment strategies through authentic patient experiences....
Published August 4, 2026
Caregiver Burden Is a Clinical Trial Risk. - Why Are We Still Ignoring It

Caregiver Burden Is a Clinical Trial Risk: Why Are We Still Ignoring It?

Patients carry the diagnosis. Caregivers often carry the burden. Clinical trials often focus on reducing burden for patients. But in many therapeutic areas, especially rare disease, pediatric, neurology, and complex...

Published August 3, 2026
What Meaningful Patient Advocacy Partnerships Look Like

What Meaningful Patient Advocacy Partnerships Look Like

The Strongest Advocacy Partnerships Begin Before There Is a Project Many biopharma companies first engage patient advocacy organizations when a specific need arises. A clinical trial needs recruitment support. A...

Published July 28, 2026
Trust - The Most Valuable Currency in Patient Advocacy and Biopharma Partnerships

Trust: The Most Valuable Currency in Patient Advocacy and Biopharma Partnerships

Trust Is Built Long Before It Is Needed Trust is often described as a cornerstone of healthcare. In patient advocacy and biopharma partnerships, it is something even more fundamental. It...

Published July 27, 2026
How Advocacy Insights Improve Trial Recruitment and Retention

How Advocacy Insights Can Improve Trial Recruitment and Retention

The Most Effective Recruitment Strategy May Not Start With Recruitment Patient advocacy organizations are often viewed as recruitment partners. Sponsors may engage them to help raise awareness, distribute study information,...

Published July 26, 2026
Why Rare Disease Recruitment Requires a Community-First Approach

Why Rare Disease Recruitment Requires a Community-First Approach

Rare Disease Recruitment Is Not a Volume Problem Many clinical trial recruitment strategies are built around reach. More awareness. More advertising. More referrals. More outreach. In common disease states, increasing...

Published July 15, 2026
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