1. Conference & Meeting Attendance
2. Development Program Update
3. Expanded Access Program
4. Stay up to date with our progress!

Conference & Meeting Attendance

Mandos Health is proud and excited to be supporting and attending several upcoming medical and patient advocacy conferences:

Ara Parseghian Medical Research Foundation (APMRF), National Niemann-Pick Disease Foundation (NNPDF), the American Neurological Association (ANA), the Child Neurology Society (CNS), and the International Niemann-Pick Disease Alliance (INPDA) meetings.

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! patientadvocacy@mandoshealth.com

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 reviewing and analyzing all possible sources of data regarding adrabetadex and NPC to determine a path forward, including clinical trials, EAP, natural history studies, and registries.

We are getting closer to finishing our assessment.

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

We plan to release a series of publications and provide presentations at these important conferences.

This will include data on the Phase 2b/3 study, our additional clinical analyses and future development plans.

We also look forward to introducing you to a new senior team member, Dr. Jonathan Yong, as SVP Medical Affairs. Jonathan previously led medical affairs for Spinraza, one of the most successful Orphan CNS drug launches. Jonathan was most recently at Cerevel.

Expanded Access Program (EAP) Update

We want to remind you that we remain steadfast in our commitment to the EAP and providing drug supply.

Recently, we invested in securing additional drug product, which allows continued supply of adrabetadex through 2025.

Mandos Health will continue to work with the clinicians, researchers and sites who continue to provide expanded access and care to NPC patients and their families in North America.

Stay up to date with our progress!

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.

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