When you look at the reference given by ATH when it was telling the effect of ATH434 on the UMRS score, it seems very likely that ATH434 works. I am almost sure that it works. Of course, it isn't easy to compare 2 different studies, but to me, it seems quite clear. Only 7 cases make another problem. The progression of the UMRS score is at its highest in early cases.

When you look at the results of this reference study it is easy to say as ATH said: "As MSA is a rapidly progressive and
unremitting disease, we expected to see decline in all participants. Instead, we saw favorable
clinical and biomarker outcomes in some patients suggesting that ATH434 has the potential to
modify the course of this devastating condition".

This is the abstract

Multicenter Study

Lancet Neurol

. 2013 Mar;12(3):264-74.

doi: 10.1016/S1474-4422(12)70327-7. Epub 2013 Feb 5.

The natural history of multiple system atrophy: a prospective European cohort study

Gregor K Wenning ¹, Felix Geser, Florian Krismer, Klaus Seppi, Susanne Duerr, Sylvia Boesch, Martin Köllensperger, Georg Goebel, Karl P Pfeiffer, Paolo Barone, Maria Teresa Pellecchia, Niall P Quinn, Vasiliki Koukouni, Clare J Fowler, Anette Schrag, Christopher J Mathias, Nir Giladi, Tanya Gurevich, Erik Dupont, Karen Ostergaard, Christer F Nilsson, Håkan Widner, Wolfgang Oertel, Karla Maria Eggert, Alberto Albanese, Francesca del Sorbo, Eduardo Tolosa, Adriana Cardozo, Günther Deuschl, Helge Hellriegel, Thomas Klockgether, Richard Dodel, Cristina Sampaio, Miguel Coelho, Ruth Djaldetti, Eldad Melamed, Thomas Gasser, Christoph Kamm, Giuseppe Meco, Carlo Colosimo, Olivier Rascol, Wassilios G Meissner, François Tison, Werner Poewe; European Multiple System Atrophy Study Group

Affiliations expand

• PMID: 23391524

PMCID: PMC3581815 DOI: 10.1016/S1474-4422(12)70327-7

Abstract

Background: Multiple system atrophy (MSA) is a fatal and still poorly understood degenerative movement disorder that is characterised by autonomic failure, cerebellar ataxia, and parkinsonism in various combinations. Here we present the final analysis of a prospective multicentre study by the European MSA Study Group to investigate the natural history of MSA.

Methods: Patients with a clinical diagnosis of MSA were recruited and followed up clinically for 2 years. Vital status was ascertained 2 years after study completion. Disease progression was assessed using the unified MSA rating scale (UMSARS), a disease-specific questionnaire that enables the semiquantitative rating of autonomic and motor impairment in patients with MSA. Additional rating methods were applied to grade global disease severity, autonomic symptoms, and quality of life. Survival was calculated using a Kaplan-Meier analysis and predictors were identified in a Cox regression model. Group differences were analysed by parametric tests and non-parametric tests as appropriate. Sample size estimates were calculated using a paired two-group t test.

Findings: 141 patients with moderately severe disease fulfilled the consensus criteria for MSA. Mean age at symptom onset was 56·2 (SD 8·4) years. Median survival from symptom onset as determined by Kaplan-Meier analysis was 9·8 years (95% CI 8·1-11·4). The parkinsonian variant of MSA (hazard ratio [HR] 2·08, 95% CI 1·09-3·97; p=0·026) and incomplete bladder emptying (HR 2·10, 1·02-4·30; p=0·044) predicted shorter survival. 24-month progression rates of UMSARS activities of daily living, motor examination, and total scores were 49% (9·4 [SD 5·9]), 74% (12·9 [8·5]), and 57% (21·9 [11·9]), respectively, relative to baseline scores. Autonomic symptom scores progressed throughout the follow-up. Shorter symptom duration at baseline (OR 0·68, 0·5-0·9; p=0·006) and absent levodopa response (OR 3·4, 1·1-10·2; p=0·03) predicted rapid UMSARS progression. Sample size estimation showed that an interventional trial with 258 patients (129 per group) would be able to detect a 30% effect size in 1-year UMSARS motor examination decline rates at 80% power.

Interpretation: Our prospective dataset provides new insights into the evolution of MSA based on a follow-up period that exceeds that of previous studies. It also represents a useful resource for patient counselling and planning of multicentre trials.


Here you find the full text because the abstract does not tell the point I want you to see table 3 and Fig. 3.

https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(12)70327-7/fulltext