Med-Life Discoveries

04/15/2026

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.

04/09/2026

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.

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...

02/28/2026

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

02/27/2026

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

02/27/2026

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.

02/27/2026

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.

02/27/2026

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!

02/19/2026

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.

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

02/10/2026

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.

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

02/05/2026

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!!

01/29/2026

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.

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

12/22/2025

From Shawn, Tara, Kaeli, and the rest of the MLD team, we want to wish everyone a safe and happy holiday season. Early in 2026 we will provide a more complete program update with tentative timelines. Our current focus is on getting a face-to-face meeting with the Agency, as they have indicated they would like to meet to discuss the overall trial design including confirmation of endpoints and details on how to safely dose escalate in patients. As you are aware, the FDA is in a state of chaos and combined with the shutdown, there have been delays in timeline across the board. The whole process continues to be slower than we would like, but all we can do is hope the FDA agrees to a productive discussion about the program and we eventually get this study going (especially since we just packaged all the drug for this study this week!!).

Our little team is as dedicated as ever to getting a safe plasmalogen therapy available for RCDP patients. We will get there! Have a great holiday season and we will be in touch in the new year.

Sincerely,

The MLD team