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EAN 2019 Brochure: Roche Neuroscience Portfolio

Brochure available at the medical booth to inform attendees about the Roche Neuroscience portfolio

EAN 2019 Infographic: Why is early diagnosis important?

Under diagnosis and delays in diagnosis represent substantial unmet needs in AD. This infographic discusses the potential benefit of earlier diagnosis and the identification of key biomarkers with potential utility in early diagnosis.

EAN Oral Presentation 2019 Design of an open-label, adaptive multiple-dose study to investigate the PK/PD of RG6042 in CSF and plasma following intrathecal administration in patients with HD

This presentation will describe the rationale and design of the GEN-PEAK study, which will collect clinical PK and PD data to characterise the CSF and plasma PK of RG6042 to further enhance the undertsanding of the most appropriate dosing regimens.

EAN 2019 Leaflet: Clinical trials in ASD

This leaflet provides an overview of both the ongoing and completed Roche clinical trials for the core symptoms of ASD including the balovaptan program and investigations into measures of repetitive and restrictive beaviours in ASD

EAN 2019 Infographic: Clinical development programmes in Alzheimer's disease: Ongoing clinical trials and biomarkers: Gantenerumab

Gantenerumab is currently being evaluated in the Phase III GRADUATE I (NCT03444870) and GRADUATE II (NCT03443973) clinical trials. Crenezumab is being investigated in the Alzheimer’s Prevention Initiative Study in Autosomal Dominant AD (ADAD). RO7105705 is currently being evaluated in the Phase II Tauriel (NCT03289143) trial for early (prodromal-to-mild) AD. Recruitment is underway for the Lauriet trial (NCT03828747) where RO7105705 will be investigated in moderate AD. This infographic contains detailed information regarding the study designs, inclusion criteria and outcome measures. The use of biomarkers throughout the AD clinical development process is also summarised.

EAN ePoster 2019 Long-Term Reduction in 48-Week Confirmed Disability Progression After 5 Years of ▼Ocrelizumab Treatment in Patients with Relapsing Multiple Sclerosis

This data expands upon the previous presentations on the effect of ▼ocrelizumab on disability progression rates from the OPERA I and OPERA II Phase III studies in patients with relapsing multiple sclerosis. These analyses indicate the effect of ocrelizumab on disability progression with 48 weeks’ confirmation was greater than on 12-week and 24-week confirmed disability progression.

EAN HD Meet the Expert 2019 “Aware of rare: What’s new in Huntington’s disease”

At the EAN conference, Roche is holding Meet-the-Expert sessions at the Roche medical booth. During these short presentations, experts will present on a topic of scientific value to the community, with the aim of raising awareness of research in the field. Importantly, after each presentation, congress attendees can ask questions to the presenter, and network with peers. Prof. Landwehrmeyer will provide an overview of Huntington’s disease and current molecules under development.

EAN ePoster 2019 RAINBOWFISH: A study of risdiplam (RG7916) in infants with pre-symptomatic spinal muscular atrophy (SMA)

RAINBOWFISH (NCT03779334) is an open-label, single-arm, multicenter clinical study to investigate the efficacy, safety, PK and PD of risdiplam in infants with genetically diagnosed SMA who are not yet presenting with symptoms. This ePoster provides an overview of the RAINBOWFISH study design and study objectives.

EAN ePoster 2019 ENSEMBLE Baseline Results and ENSEMBLE PLUS Study Design: An Investigation of Shorter ▼Ocrelizumab Infusion Time in Patients with Relapsing-Remitting Multiple Sclerosis_Supplemental Material

Supplemental material for EAN_ePoster_2019_ENSEMBLE Baseline Results and ENSEMBLE PLUS Study Design: An Investigation of Shorter ▼Ocrelizumab Infusion Time in Patients with Relapsing-Remitting Multiple Sclerosis

AAN Presentation 2019: Update from SUNFISH part 1: Safety, tolerability and PK/PD from the dose-finding study, including exploratory efficacy data in patients with Type 2 or 3 spinal muscular atrophy (SMA) treated with risdiplam (RG7916)

SUNFISH (NCT02908685) is a multicenter, two-part, randomized, placebo-controlled, double-blind Phase 2 study evaluating the efficacy and safety of risdiplam (RG7916) in patients with Type 2 or Type 3 SMA aged 2-25 years. This presentation describes safety, tolerability and PK/PD data from all patients in SUNFISH Part 1 and exploratory efficacy data in patients treated with risidplam for ≥1 year.

AAN Presentation 2019: Translational pharmacokinetic/pharmacodynamic (PK/PD) modeling strategy to support RG6042 dose selection in Huntington’s disease (HD)

This presentation will summarise the development of a pharmacokinetic/pharmacodynamic (PK/PD) model for the antisense oligonucleotide RG6042 to enable dose selection for the GENERATION HD1 Phase III trial. Interim results from an open-label extension to the RG6042 Phase I/IIa study have added to our understanding of the PK/PD profile of RG6042.

AAN Presentation 2019: FIREFISH Part 1: Survival, ventilation and swallowing ability in infants with Type 1 SMA receiving risdiplam (RG7916)

FIREFISH (NCT02913482) is an ongoing, multicenter, open-label study aiming to assess the safety and efficacy of risdiplam (RG7916) in infants aged 1–7 months with Type 1 SMA and two SMN2 gene copies. This presentation describes one-year data on event-free survival rates and swallowing ability in infants from FIREFISH Part 1.

CHORDS sheds light on safety and effectiveness of ocrelizumab in real-world setting

Thomas Leist takes us through the 1-year interim analysis of the CHORDS study of ocrelizumab comprising multiple sclerosis patients drawn from community practice.

AAN Poster 2019: Preliminary reliability and validity of a novel digital biomarker smartphone application to assess cognitive and motor symptoms in Huntington’s disease (HD)

This study aimed to determine the relability and validity of a remote digital monitoring platform comprised of a smartphone application to measure cognitive and motor symptoms in patients with early manifest Huntington's disease.

Risdiplam potential for type 2, 3 SMA demonstrated

Laurent Servais presents the safety and exploratory efficacy data from the SUNFISH study evaluating the SMN2 pre‐mRNA splicing modifier risdiplam in individuals who have type 2 or 3 spinal muscular atrophy.

Paediatric MS disability scoring system developed

Jonathan Santoro summarises their research evaluating disability distribution in paediatric-onset multiple sclerosis using a novel scoring model, and discusses the implications for the care of patients.

AAN Poster 2019 VERISMO: A Post-Marketing Safety Study to Determine the Incidence of All Malignancies and Breast Cancer in Patients With Multiple Sclerosis Treated With ▼Ocrelizumab

This presentation describes the study-design of the post-marketing safety study VERISMO, which will increase understanding of any potential risk of malignancy/breast cancer in ▼ocrelizumab-treated patients with MS.

Amyloid PET may predict cognitive decline in patients with MS

Jordi Matias-Guiu summarises the findings of their prospective study investigating the use of amyloid positron emission tomography to identify multiple sclerosis patients likely to experience cognitive impairment.

ESCAP 2019 poster: Psychometric Properties of a Novel Vineland-II 2-Domain Composite Score to Assess Social Communication and Social Interaction in Autism Spectrum Disorder

The Vineland™-II 2-domain composite (2DC) score is calculated as the arithmetic mean of the Vineland™-II Socialization and Communication domain scores. In this study, a psychometric analysis of this new scale was carried out using data from the VANILLA phase 2 study of balovaptan.

ESCAP 2019 presentation: Effects of Balovaptan on Health-related Quality of Life of Adult Males with Autism Spectrum Disorder: Results from a Phase 2 Randomized Double-Blind Placebo Controlled Study (VANILLA)

VANILLA was a 12-week, phase 2, randomized study that assessed the efficacy and safety of balovaptan in adult men with ASD. The health-related quality of life (HRQoL) of the participants was an exploratory objective of the study.

CHDI 2019 poster: Disease progression modeling in early Huntington’s disease (HD)

This poster presents models used to characterise disease progression in early HD clinical trials based on cUHDRS that have been developed from the 4-year observational TRACK-HD and Enroll-HD study databases.

CHDI 2019 poster: Positron emission tomography (PET) biodistribution study and radiation dosimetry of a radiolabeled HTT-ASO after intrathecal (IT) administration in rats

This study conducted in rats assessed the feasibility of PET imaging to track the distribution and uptake of intrathecally administered radiolabelled RG6042, an antisense oligonucleotide drug candidate for HD

CHDI 2019 poster: Resting state electroencephalography (EEG) biomarkers in Huntington’s disease (HD): Feasibility, reliability and relation to HD pathology

This study assessed the potential of resting-state-EEG-derived measures as biomarkers for HD using baseline resting-state EEG acquired in 46 participants of a Phase I/IIa study in early manifest HD (Stage 1).

CureSMA Conceptual Modelling Poster 2019:Qualitative research to explore the patient and caregiver-reported experience of symptoms and impacts in spinal muscular atrophy (SMA) Types 2 and 3 - the development of a conceptual model

Spinal muscular atrophy (SMA) can have a significant effect on an individual’s Health Related Quality of Life and has a substantial impact to patients’, caregivers’ and family members’ physical, emotional, psychological and social well-being. This poster describes the development of a conceptual model to better understand the patient and caregiver experience of SMA.

CureSMA SUNFISH Presentation 2019: Update from SUNFISH Part 1: Safety, tolerability and PK/PD from the dose-finding study, including exploratory efficacy data, in patients with type 2 or 3 spinal muscular atrophy (SMA) treated with risdiplam (RG7916)

SUNFISH (NCT02908685) is a multicenter, two-part, randomized, placebo-controlled, double-blind study assessing the safety, tolerability, PK and PD of risdiplam in patients with Type 2 or Type 3 spinal muscular atrophy, aged 2–25 years of age. This presentation describes safety, tolerability, PK/PD and exploratory effiacy data from SUNFISH Part 1 patients who have recieved risdiplam for a minimum of 12 months.

CureSMA JEWELFISH Poster 2019: JEWELFISH: Risdiplam (RG7916) increased survival of motor neuron (SMN) protein levels in patients with spinal muscular atrophy (SMA) that have previously received therapies targeting SMN2 splicing, or olesoxime

JEWELFISH (NCT03032172) is an ongoing, multicenter, open-label study to assess the safety, tolerability and PK/PD relationship of daily, oral risdiplam in adults, children and infants with SMA previously enrolled in Study BP29420 (MOONFISH) with the splicing modifier RO6885247 or previously treated with SPINRAZA® (nusinersen), olesoxime or ZOLGENSMA® (AVXS-101). This presentation provides an overview of the latest JEWELFISH study enrollment criteria and describes the safety and PK/PD of risdiplam in 12 patients.

CureSMA Rasch Mapping Poster 2019: Using Rasch analysis to estimate thresholds associated with gain/loss of daily function on the Motor Function Measure (MFM)

Rasch analysis is a statistical method that can predict an individual's probable progression on clinical scales based on their level of function on an underlying disease spectrum. This poster presentation uses the Rasch method to estimate the threshold at which a daily function is gained/lost on the MFM32 scale.