Anyone have experience with Multiple System Atrophy (MSA)?

Below is MSA pharmaceutical clinical testing trial info, sent to a friend dealing with MSA. Hope it helps all.

Current pharmaceutical clinical testing for Multiple System Atrophy (MSA) is heavily focused on disease-modifying therapies, specifically targeting the reduction of
-synuclein protein aggregation, which is believed to drive disease progression. As of early 2026, the field has seen several key developments, including Phase 2/3 trials for investigational drugs and FDA Fast Track designations.
National Institutes of Health (.gov)National Institutes of Health (.gov) +4
Here are the key ongoing and recent clinical trials and therapeutic candidates:
Top Clinical Trial Candidates (2025–2026)
Amlenetug (Lu AF82422 / Lundbeck): This is a human monoclonal antibody designed to bind to
-synuclein and inhibit its aggregation.
Status: A Phase 3 trial, MASCOT (NCT06706622), was initiated in late 2024/early 2025 following positive results from the Phase 2 AMULET trial. It was granted FDA Fast Track designation in February 2025.
Emrusolmin (TEV-56286 / MODAG / Teva): A small molecule designed to target
-synuclein oligomers.
Status: In Phase 2 development (TOPAS-MSA study). It was granted FDA Fast Track and Orphan Drug designation in September 2025.
ATH434 (Alterity Therapeutics): An oral iron-targeting agent designed to reduce iron-induced
-synuclein aggregation in the brain.
Status: Reported positive Phase 2 results in early 2025, demonstrating reduced brain atrophy.
AB-1005 (AskBio / Bayer): An investigational gene therapy (AAV2-GDNF) delivered to the brain to promote the survival of dopaminergic neurons.
Status: Completed enrollment for the Phase 1 REGENERATE MSA-101 trial in September 2025.
Ampreloxetine (Theravance Biopharma): A norepinephrine reuptake inhibitor focusing on symptomatic treatment of neurogenic orthostatic hypotension (nOH) in MSA.
Status: Active, but reported in March 2026 that the Phase 3 CYPRESS study did not meet its primary endpoint.
ION464 (Ionis Pharmaceuticals): An antisense oligonucleotide (ASO) designed to prevent the production of
-synuclein protein.
AskBioAskBio +10
Key Areas of Investigation

-Synuclein Reduction: Antibodies (amlenetug) or small molecules (emrusolmin) to clear toxic protein buildup.
Neuroprotection/Iron Regulation: Agents like ATH434 to prevent brain atrophy.
Gene Therapy: Using GDNF to protect cells.
Symptomatic Management: Treating debilitating orthostatic hypotension (e.g., ampreloxetine).
Natural History Studies: Studies such as TRACK-MSA (NCT04450992) are ongoing to identify better outcome measures for future trials.
NYU Grossman School of MedicineNYU Grossman School of Medicine +3
Recent Trial Outcomes
Sirolimus: A study supported by the NIH was ended early after interim analysis indicated it was not effective in slowing MSA progression.
Trial Challenges: The rapid progression of MSA makes trial recruitment and retention challenging, with a high failure rate for neuroprotective compounds.
National Institutes of Health (.gov)National Institutes of Health (.gov) +3
Regards,
Sagan

@hopeful33250

Of course I would invite anyone with this disease to share or ask questions about MSA. I learned a lot about it as we didn't have much help here in Maine. I found my Joe had adverse reactions to medications, as do I. As an example, for swelling, I would feed him pineapple.

Here’s info on the test they did at UCSF Movement Center.
The patient returns today for the Syn-One skin biopsy. We discussed the main benefit of the procedure would be to look for the absence or presence of phosphorylated alpha-synuclein. The presence of this protein encompasses a group of neurodegenerative diseases that includes Parkinson's Disease, Dementia with Lewy bodies, Multiple System Atrophy, Pure Autonomic Failure and REM sleep behavior disorder. The presence of this abnormal protein in cutaneous nerve fibers is highly specific for a diagnosis of synucleinopathy but can not currently distinguish between the different synucleinopathies. In addition to testing for phosphorylated alpha-synuclein, the test evaluates for reduced intraepidermal nerve fiber density, amyloid deposition and dermatological abnormalities. The pattern of distribution, as well as the intraepidermal nerve fiber density, may help provide some added insights in helping differentiate between the different synucleinopathies but this remains an area of continued research.
I should get the results back in 4-5 weeks from a lab in Arizona.

MSA has been a discussion my doctors have been having about my health. I will be very interested in the results you get. I have read about the protein that predisposes one to MSA. No tests done yet on me. Nephrologist says she can sometimes see the protein on tests she has run but it hasn’t shown up to date.

My skin biopsies were sent to a lab in Arizona. I believe they are the only lab in US running this test. I could be wrong.

J finally got back my Syn-One skin biopsy and spoke to my Neurologist at UCSF. 2 of the 3 biopsies tested positive for MSA. It seems I have MSA-C. It's good to find out after all these years and doctors, to finally know what's going on. Now I just have to deal with the symptoms the best I can.

@scottmoon

I appreciate the update on the results of the Syn-One skin biopsy.

What are the most troublesome symptoms you are experiencing? Have you been offered any suggestions in dealing with the symptoms?

My worst symptoms are balance issues, incontinence, exhaustion, Blood pressure swings, and speaking problems.
My Neurologist wants to try to get my BP issues under control.

@scottmoon,

You mentioned speaking problems. Is this word finding problems or pronunciation issues? Has speech therapy ever been suggested?