1. Rare Disease Day 2025
2. Development Program Update
3. Expanded Access Program
4. Community Update

Rare Disease Day

Mandos Health is proud to support Rare Disease Day, a global initiative to raise awareness and generate support for everyone who is on a rare medical journey. It takes place on the last day of February, which this year is February 28th.

Did you know, the date was chosen because in leap years, it is February 29, the rarest date?

We will be highlighting social media from our NPC and rare disease communities such as NNPDF, INPDA, NPUK, Firefly Fund, NORD, and EveryLife Foundation.

Internally, our team members will be sharing random acts of kindness they have done in the past few months…

Every day we support the NPC and broader rare disease community by advancing research and building relationships with patients, their families, clinicians, policy makers, and everyone striving for care and novel treatment that aligns with patients, accessible to all.

Development Program Update

At Mandos Health, our goal is to move adrabetadex forward in its development in the most efficient manner possible and in consideration of all those suffering with NPC1.

We are grateful for the FDA’s thoughtful collaboration on the development program.

We will be releasing data over the course of the year from our analysis and are excited to share this soon. This will include data on the Phase 2b/3 study, our additional clinical analyses and future development plans.

Expanded Access Program (EAP) Update

At the start of 2025 we want to remind you that we remain steadfast in our commitment to the EAP.

Mandos Health is grateful to our partners and continues to work with the clinicians, researchers and sites who provide expanded access and care to the NPC patients and their families.

Community Update

Mandos Health will be attending many conferences in 2025 including:

World Symposium, the American Academy of Neurology (AAN), Ara Parseghian Medical Research Foundation (APMRF), National Niemann-Pick Disease Foundation (NNPDF), the American Neurological Association (ANA), and the International Niemann-Pick Disease Alliance (INPDA) meeting.

We enjoy engaging all stakeholders with our News, conference attendance, and patient advocacy organization meetings. Please contact us if you would like to get together at any of these conferences!

Stay up to date with our progress!

As always, we know every day matters.

We couldn’t be more grateful for the trust you continue to place in us.

Stay up to date with us at www.mandoshealth.com