Med-Life Discoveries

Our Executive VP, Innovative Therapies has been busy the last couple days learning about innovation in rare disease drug development at the Rare Disease Scientific Symposium.

In this edition of the literature blog, we are highlighting a paper that discusses the relationship between APOE status and plasmalogen deficiency with regards to the risk for Alzheimer's disease.

Plasmalogen deficiency and the Alzheimer’s disease risk of apolipoprotein ɛ4 — MED-LIFE DISCOVERIES Hällqvist J, Taanman J, Göteson A, Heywood WE, Schott JM, Hardy J, Landén M, Zetterberg H, Mills K, and Ginsberg L. (2026) Plasmalogen deficiency and the Alzheimer’s disease risk of apolipoprotein ɛ4. Brain Communications The apolipoprotein E gene ( APOE ) encodes the apoE protein and is respo...

MLD is proud to be a part of the rare disease community and happy to help raise awareness today for Rare Disease Day!

The majority of rare diseases are genetic and, of these, 70% begin in childhood.

With tomorrow being Rare Disease Day, here's another fact: out of all rare diseases, 72% have a genetic cause. The other 28% of rare diseases are the result of bacteria or viral infections, allergies, environmental causes, or are a rare cancer.

Tomorrow is Rare Disease Day!
Although each rare disease affects a small group of people - sometimes an incredibly small group - when taken together, 5% of the world's population is affected by a rare disease.

February 28th is Rare Disease Day, a day to help raise awareness of rare diseases. Although each are considered rare, there are over 6000 rare diseases that have been identified and more are discovered every year!

Just a quick reminder that our virtual update for RCDP families is on February 24th and registration for the update closes tomorrow at 5pm EST. Click the link below for more information and to register.

Virtual Update Registration — RhizoTRIAL.org Hello Rhizo Families, Thank you to the Rhizo community for your continued support. We know that there have been a number of requests for an update on the clinical trial and the MLD program overall. We are going to be holding a virtual update in the week leading up to Rare Disease Day to answer the

We know a virtual update has been long awaited by the RCDP families, so we are excited to announce that we have one planned the week leading up to Rare Disease Day on Tuesday, February 24th at 5pm ET. Registration is required to attend the update, so if you are interested, please click the link below for more information and to register. Registration will close at 5pm ET on Friday, February 20th.

Virtual Update Registration — RhizoTRIAL.org Hello Rhizo Families, Thank you to the Rhizo community for your continued support. We know that there have been a number of requests for an update on the clinical trial and the MLD program overall. We are going to be holding a virtual update in the week leading up to Rare Disease Day to answer the

We are finished up another week of monitoring the RCDP natural history study records here at Nemours Children’s Hospital in Wilmington Delaware. Such a beautiful facility and always so willing to welcome us. Thanks to all the RCDP families that keep attending their clinical study visits. This program is a success because of you!!

This year we are starting off the literature blog with a paper that discusses the effects of aging on the lipidome of the lateral ventricle choroid plexus, the main site of cerebrospinal fluid production.

The lipidome of the lateral ventricle choroid plexus exhibits sex-specific changes across aging — MED-LIFE DISCOVERIES Loupiac G, Andriambelo B, Avila-Lopez J, Vachon A, Avino M, Ahumada JR, Miard S, Picard F, Qiu Y, Kurland IJ, Laurent B, and Plourde M. (2025). The lipidome of the lateral ventricle choroid plexus exhibits sex-specific changes across aging. Journal of Neurochemistry