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A Phase 2 study to evaluate the safety and efficacy of prasinezumab in early Parkinson's disease (PASADENA): Rationale, design and baseline data

Prasinezumab is a humanised, monoclonal IgG1 antibody designed to target α-synuclein. PASADENA is the ongoing Phase 2 multicentre, randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of intravenous prasinezumab, administered every 4 weeks in patients with early-stage PD. The primary endpoint for the study is the change in MDS-UPDRS total score (sum of Parts 1, 2 and 3) that includes motor and non-motor assessments. Baseline patient demographic and disease characteristic data shows that the PASADENA population is similar to the Parkinson's Progression Markers Initiative (PPMI) study population and is, therefore, representative of a wider PD population.

Blood Neurofilament Light Chain Levels And Association With Brain Volume Change In Patients With Ppms And Rms Before And Under Treatment With Ocrelizumab▼

Blood NfL is a biomarker of neuroaxonal injury in MS and correlates with CSF levels and clinical and MRI measures of disease activity. Here we assess whether NfL levels measured before and during OCR▼ treatment were associated with BV changes in patients with PPMS and RMS.

Long-Term Reduction of Relapse Rate and Confirmed Disability Progression After 6 Years of Ocrelizumab▼ Treatment in Patients With Relapsing Multiple Sclerosis

Adherence to disease modifying therapy is critical for achieving therapeutic goals in MS. This analysis evaluated persistence with and adherence to ocrelizumab▼ compared with injectable, oral, and other intravenous DMTs in a real-world, commercially insured population.

Efficacy of Satralizumab Monotherapy in Prespecified Subgroups of SAkuraStar, a Phase 3 Study in Patients With Neuromyelitis Optica Spectrum Disorder

This analysis evaluated the efficacy of satralizumab monotherapy vs placebo for prevention of protocol-defined relapse in predefined subgroups of the SAkuraStar study, including patients with NMO/NMOSD who were postive for aquaporin-4.

Preliminary results from a 15-month open-label extension (OLE) study investigating RG6042 huntingtin protein (HTT) antisense oligonucleotide (ASO) in adults with manifest Huntington’s disease (HD)

Long-term safety, tolerability and biomarker effects of tominersen (most recently known as RG6042) were evaluated in participants of the 15-month open-label extension of the Phase I/IIa study. Results of this preliminary 15-month analysis will be presented.

Remote and frequent assessment of Huntington’s disease (HD) in clinical trials: Strategies for assessing and accounting for the practice effect

The improvement in performance over time resulting from the repetition of a task is known as practice effect. This study assessed the impact of practice effects on performance in individuals with HD during digital testing sessions and established the number of practice test iterations required to accurately estimate true performance changes.

The Digital-HD study: Smartphone-based remote testing to assess cognitive and motor symptoms in Huntington’s disease (HD)

This study evaluated the feasibility and acceptability of smartphone-based remote testing and its ability to meaningfully quantify signs, symptoms and impairments in participants in the Digital-HD study. Differences in performance of individuals with premanifest Huntington’s disease (HD), manifest HD and healthy controls, were compared across a series of predefined digital tests.

Shorter door-to-needle times of intravenous alteplase improve “efficiency” of care and moderately affect “quality” of care: Study based on a large comprehensive stroke center

Quicker IV alteplase treatments resulted in improved clinical outcomes than treatment within 60 minutes.

Assessment of Current Trends in Alteplase Use in the Treatment of Cardiovascular Diseases

AIS is the most common indication for current alteplase use. Differences in payment amd rurality may have resulted from differing levels of access to care.

SMA Europe Poster 2020 FIREFISH: Safety, survival and motor function in infants with Type 1 spinal muscular atrophy (SMA) receiving risdiplam (RG7916)

FIREFISH (NCT02913482) is an ongoing, multicenter, open-label study of risdiplam (RG7916) in infants aged 1–7 months with Type 1 SMA and two SMN2 gene copies. Part 1 is assessesing the safety, tolerability, PK and PD of different risdiplam dose levels; confirmatory Part 2 is assessesing the safety and efficacy of risdiplam. This poster presentation provides an update on the latest safety and efficacy data from FIREFISH Part 1 in infants who have received treatment with risdiplam for 16 months.

SMA Europe Poster 2020 JEWELFISH: Safety and pharmacodynamic data in patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam (RG7916) that have previously been treated with nusinersen

JEWELFISH (NCT03032172) is an ongoing, multicenter, open-label study assessing the safety, tolerability and PK/PD relationship of daily oral risdiplam in non-naïve patients with SMA, aged 6 months to 60 years. This poster presentation provides an update on the latest available safety and PD data.

SMA Europe Poster 2020: Pooled safety data from the risdiplam (RG7916) clinical trial development program

FIREFISH (NCT02913482) aims to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of risdiplam in infants with Type 1 SMA aged 1–7 months. SUNFISH (NCT02908685) aims to evaluate the safety, tolerability, PK, PD and efficacy of risdiplam in patients with Type 2 or 3 SMA aged 2–25 years. JEWELFISH (NCT03032172) aims to assess the safety, tolerability, PK and PD of risdiplam in in non-naïve patients with SMA, aged 6 months to 60 years. This poster presentation will include pooled safety analyses from FIREFISH Parts 1 and 2, SUNFISH Part 1 and JEWELFISH. This analysis will add to the understanding of the longer-term safety profile of risdiplam in individuals with SMA.