Patient Advocacy Strategies

12/16/2025

A Historic Win for Rare Disease—But a Warning for Public Health Policy

Today is a day of celebration. Metachromatic Leukodystrophy (MLD) and Duchenne Muscular Dystrophy (DMD) have officially been added to the federal Recommended Uniform Screening Panel (RUSP).

For years, the MLD and DMD communities have fought for this. In April 2025, when the federal advisory committee (ACHDNC) was suddenly disbanded, their path to screening was effectively blocked.

They didn’t wait for the system to fix itself. By taking a novel approach - publishing their evidence reviews, presenting the evidence in cooperation with the EveryLife Foundation and using that evidence to appeal directly to HHS, these advocates secured a victory that will save thousands of lives. In a year of massive upheaval, this is a testament to the power of patient advocacy.

The Impact:

Implementation: We expect all states to screen for these conditions within 5 years.

Early Action: Many states will move within the next 3 years.

Life-Saving Outcomes: Early detection is the only way to access time-sensitive treatments for MLD and DMD.

But we must be honest: This is a "Plan B" strategy, not a sustainable policy.

We cannot use direct appeals to the HHS Secretary for future conditions. To protect the integrity of Newborn Screening, we need an evidence-based approach, not a political one.

The missing piece? The reauthorization of the Newborn Screening Saves Lives Act.

We need to reinstate a formal federal advisory committee to ensure that every condition is evaluated by scientists and clinicians—not caught in a political vacuum.

At Patient Advocacy Strategies, we are proud to stand with these communities. We are ready to help your organization navigate this new, complex landscape and build a state-by-state strategy that ensures no child is left behind.

Let’s celebrate the win today, and get back to work tomorrow.



https://patientadvocacystrategies.com/blog/news/mld-dmd-added-to-the-federal-rusp-a-victory-for-newborn-screening/

10/22/2025

PAS's Lesa Brackbill, Author, and Argall will be attending the American College of Rheumatology Convergence in Chicago from Oct. 25 - 28.

We look forward to speaking with you, gathering new insights, and connecting with others within the ACR community. Share in the comments if you'll be there – we would love to connect!

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09/30/2025

“No child should die or suffer disabilities if a simple bloodspot can prevent it.” - Dr. Guthrie

If it weren't for Dr. Guthrie's persistence in developing a way to test babies for PKU in order to treat them as early as possible, we would not have as it is today.

We are grateful for all of the stakeholders who make up the Newborn Screening system - scientists, clinicians, public health programs, geneticists, specialists, and drug developers.

And we are extra grateful for all the family advocates who work toward the change needed to save lives.

09/29/2025

No matter how the diagnosis occurs, the child will still have the disease, and the family will adjust to the new reality. The difference is that a diagnosis through Newborn Screening gives options that don’t always exist after a diagnostic odyssey. Newborn Screening brings diagnosis with hope. - Lesa Brackbill, Rare Mom and Advocate

As Awareness Month continues, our Director of Advocacy highlights the power of newborn screening for . It provides families with early diagnosis, hope, and a range of options.

This is a great example of how industry and advocacy can partner to make a real impact. PAS is here to help with your NBS strategy.

09/25/2025

"There are so many problems to solve in rare disease, but it all starts with listening to the patients; letting them know their story, their pain, their loss matters.”

PAS’s Director of Advocacy, Lesa Brackbill, understands the pain and perspective of the rare disease community, having lost her daughter, Tori, to Krabbe disease.

In the ten years since, Lesa has become a fierce advocate for newborn screening and speaking on behalf of rare disease families.

In her role at PAS, Lesa helps rare patients tell their stories so those within the rare, medical, and life sciences communities are aware of and can take action to fight back against rare illnesses.

09/22/2025

When it comes to rare, childhood conditions, early diagnosis is critical. At Patient Advocacy Strategies, we champion policies that make it happen, like . Our advocacy team, led by a rare mom and newborn screening expert, is dedicated to ensuring patients have a voice and access to the treatments they need.

Learn how we can help your company or advocacy group - link in bio.

09/19/2025

September is Newborn Screening Awareness Month!

Newborn screenings, which include blood spot tests, heart screenings, and hearing screenings, are crucial for a baby's health. The goal of these tests is to identify serious but treatable health conditions early, often before any symptoms appear.

Early detection allows for immediate intervention, which can prevent death, disability, and long-term health problems.

Each state’s Newborn Screening system operates differently and screens for different numbers of conditions.

Our team at Patient Advocacy Strategies can provide expertise to guide you through the complexity of the system and help ensure that every baby is screened for as many conditions as possible.

09/17/2025

"The work we do creates hope for patients, and that hope is paramount to the success of biotech companies."

Mike Walsh, PAS founder and CEO, shares how giving patients a voice creates hope and can be a catalyst for change.

Contact us: https://patientadvocacystrategies.com/contact/?utm_source=linkedin&utm_medium=link&utm_id=storytelling

09/17/2025

Gene therapy is saving lives but most of the patients cannot afford the price of $2 to $3 million dollars. These gene therapies shouldn’t just be for the rich or a luxury. It is time for insurance company models to change so everyone can get these lifesaving gene therapies if they require them.



Read on here

It can cure deadly diseases, save long-term healthcare costs and transform lives. But the U.S. insurance system still isn’t ready to pay for it.

09/15/2025

Diversity is critical in cancer clinical trials, however reaching diverse populations for these trials can be a challenge.

New technologies such as digital monitoring, telehealth, and home-based testing are making trials more accessible.

Representation is the first step towards equitable cancer care.

Read on here

Kimberly (Cary) Demirhan discusses the need for more diversity and inclusivity in clinical trials for patients with cancer.

09/09/2025

Significant changes in medical education: According to a recent Do No Harm report, three major healthcare accreditors still enforce DEI regulations, while seven have pulled down or ended them. Is healthcare training returning to merit-based standards?

Learn more here:

Today, Do No Harm published a new report titled “Are Medical And Healthcare Education Accreditors Abandoning DEI?” The report, a follow-up to Do No Harm’s previous report exposing accreditors for injecting identity politics into medical and healthcare education, found that the accreditation la...

07/30/2025

Patient-centricity is the way of the future for clinical trials, not just a trendy term. This Technology Networks piece analyzes how digital biomarkers are gathered via smartphones, wearables, and remote monitoring devices which are helping more individualized, easily accessible, and effective clinical research.

Learn more on how technology is speeding up therapeutic advancements and changing patient participation here.

Explore how digital biomarkers are transforming clinical trials with real-time data, continuous monitoring and AI-driven patient insights.