I am a healthcare professional

Notice


Welcome to MEDICALLY. This website is a non-promotional international resource intended to facilitate transparent scientific exchange regarding developments in medical research and disease management. It is intended for healthcare professionals outside the United Kingdom (UK) and Australia. The content on this website may include scientific information about experimental or investigational compounds, indications and services that are not approved or valid in your jurisdiction. Registration status and prescribing information of medicinal products may differ between countries. Roche and Genentech do not support, endorse or recommend the unapproved use of any compound or service in your jurisdiction, including those discussed on this website.


Please refer to local product information for any medicinal products mentioned. Information available on this website does not constitute professional medical advice, and Roche and Genentech accept no responsibility for access to or use of the same. Healthcare professionals outside the US are required to register and log-in to access the full range of content available on this website.


By clicking on one of the healthcare professional buttons below, you acknowledge you have read and understood this message and that you are requesting access to MEDICALLY. If you are not a healthcare professional, please use the other links below to access information relevant to you.


Cookies


We use cookies on this site to enable the site to function properly and to enhance your user experience. Cookies are files stored in your browser, which most websites use to personalize your web experience. Your information will only be used to provide information that is relevant to you. It will not be used for any other purpose. If you wish to restrict or block cookies, which are set on your device, then you can do this through your browser settings.


You can find out more about cookies by browsing our Privacy Policy.

 

I am a healthcare professional in Switzerland

Required field.

For healthcare professionals in Another Country browse medically.roche.com.

Not a healthcare professional? For individuals outside the US, browse Roche's patient website or roche.com. For individuals in the US, browse gene.com.

External site

By following this link, you are leaving Medically and entering a website that is not owned or controlled by Roche. Roche does not take any responsibility for access to or use of this website, nor for any content therein.

Leave site
Home Neuroscience Huntington's Disease

Huntington's Disease

What's new

Sign up or login to unlock the full suite of MEDICALLY features
Feb 27 / Roche and Genentech
Allele-selective lowering in HD: From bench to bedside
In this presentation we will share for the first time preclinical data from our allele-selective huntingtin-lowering program, HTT SNP ASO. In order to accurately identify individuals who could benefit from this approach, we have developed a novel SNP phasing assay capable of identifying distant intronic and exonic SNPs in phase with the expanded CAG mutation, paving the way for identifying multiple SNPs in parallel.
Neuroscience Huntington's Disease CHDI-2025

Sign up or login to unlock the full suite of MEDICALLY features
Feb 26 / Roche and Genentech
Development of a long-read phasing assay for allele-specific patient inclusion for Roche HTT SNP ASO program
This poster describes the development of an innovative assay that allows phasing of distant intronic SNPs with the expanded CAG repeat that causes Huntington’s disease (HD). The assay is a prerequisite for selecting individuals with HD who could benefit most from allele-selective antisense oligonucleotide treatment.
Neuroscience Huntington's Disease CHDI-2025

Sign up or login to unlock the full suite of MEDICALLY features
Feb 24 / Roche and Genentech
Towards disease modifying treatments in HD: An update from Roche
In this presentation we share Roche's and Spark's commitment in HD which expand from therapeutic development of diverse approaches to tacke HD to efforts to contribute more broadly to the HD community and advance scientific knowledge and understanding of the pathophysiology of the disease. These range from running observational studies to pioneering data sharing and harmonisation efforts in partnership with HD-RSC and CHDI.
Neuroscience Huntington's Disease CHDI-2025

Sign up or login to unlock the full suite of MEDICALLY features
Nov 8 / Roche and Genentech
Clinical Development Update on Tominersen: Fluid biomarker data from GENERATION HD1 and what it means for GENERATION HD2
This presentation includes the latest CSF fluid biomarkers results from GENERATION HD1 (YKL-40, total Tau (& ptau-181) & GFAP), related to neuroinflammation, neuronal death and astrocytic reactivity in HD. In GENERATION HD1 elevations in these biomarkers are avoided at the lowest exposures. The exposure group targeted in GENERATION HD2 has a favourable CSF fluid biomarker profile with NfL, YKL-40, total Tau & pTau-181 trending below placebo. In additiion, we provide an update on the iDMC recommendation to continue with the GENERATION HD2 study as planned after revieweing blinded data on safety, clinical, MRI & plasma NfL from 219 participants, with up to 5 doses of study treatment and an average time on treatment of 166 days.
Neuroscience Huntington's Disease HSG-2024

Sign up or login to unlock the full suite of MEDICALLY features
Nov 7 / Spark
Advancing SPK-10001 into a First-in-Human study
This presentation includes pre-clinical data describing SPK-10001, along with a high level overview of the Phase 1/2 trial evaluting the safety and efficacy of SPK-10001.
Neuroscience Huntington's Disease HSG-2024

Sign up or login to unlock the full suite of MEDICALLY features
Nov 6 / Spark
SPK-10001 AAV-based microRNA mediates non-allele specific reduction of HTT mRNA through RNA interference, demonstrating its potential for further preclinical development
SPK-10001 is an engineered adeno-associated virus expressing an artificial microRNA which targets the human HTT mRNA for degradation. The associated study determines the pharmacologic properties of SPK-10001.
Neuroscience Huntington's Disease HSG-2024
Upcoming Congresses
Access to Roche and Genentech's medical information
View related congresses

Ask a question or share feedback

Get in touch

This website is intended for healthcare professionals outside the United Kingdom (UK) and Australia. Registration status and prescribing information of medicinal products may differ between countries. Please refer to local product information for any medicinal products mentioned on this website.