Updates from Roche on Phase II Generation HD2 study
For those of you who were unable to attend the Webinar on the Phase II Generation HD2 study, here is the complete video.
👥 In this video, you will be able to listen to the updates on this study from the Clinical Science Leader from #Roche, Dr. Peter McColgan, and the Principal Investigator in Madrid (Spain), Dr. José Luis López-Sendón.
HD-CAB One Global Voice
We celebrate the first year of the HD-CAB initiative, a partnership between the European Huntington, HDYO (Huntington’s Disease Youth Organisation) and the International Huntington Association
🙌 The main mission of the HD-CAB (Community Advisory Board) is to represent the voice of the global HD community and provide the unique expertise of living with HD to stakeholders relevant for therapeutic development.
The Worm Lab during the Second Moving Forward meeting in Spain
Webinar on the updates of Wave's trial SELECT-HD
After Wave’s positive announcements last September on the Phase 1/2 SELECT-HD trial, we are excited to learn more about the last updates of the study in our upcoming webinar next Thursday 26th, January.
Dr. Ralf Reilmann, founder of the George-Huntington Institute in Muenster, will be presenting an overview of the SELECT-HD trial, which is looking at the safety of the investigational molecule called WVE-003. According to Dr Reilmann “preliminary data suggest WVE-003 is working as intended, reducing the levels of mutant huntingtin while avoiding targeting the healthy protein”.
There will be time for comments and questions after Dr Reilmann’s presentation, in our Q&A session leaded by Danlin Xu, HD Medical Director at Wave, and Marissa Volpe, HD Program Lead at Wave.
January 26th, 2023
Updates from Roche on Phase II Generation HD2 study
The GENERATION HD2 clinical trial is just about to start recruiting participants in the first of a total of 15 countries and 9 of them are in Europe: Denmark, France, Germany, UK, Poland, Italy, Spain, Portugal and Switzerland. The study aims to enroll 360 participants and evaluate the safety of the investigational drug tominersen in people with early manifest HD.
The Clinical Science Leader from Roche, Dr. Peter McColgan, and the Principal Investigator in Madrid (Spain), Dr. José Luis López-Sendón, will provide the latest updates of the study and their research efforts in HD. After the presentations, there will be time for comments and questions.
January 19th, 2023
Are you ready for a challenge? SOM3355 Trial
We would greatly appreciate the help of the European HD community in making a joint action in the recruitment of the SOM3355 trial. Efficient and timely recruitment equals faster results. Currently in Europe the only course of treatment for chorea is Tetrabenazine which has several negative side effects and limited efficacy for most patients. SOM3355, as a drug has been used for many years for a different disease. Positive results can hopefully lead to faster approval.
THE 'VOICE OF HD' NEWSLETTER IS OUT NOW!
For the 2022 Christmas Newsletter edition, we speak about the Moving Forward new project resources, the HD communication group campaigns, the upcoming EHA Conference in Belgium 2023 and our special new challenge for the HD community! Find out more here
The European Huntington Association send a Newsletter every 6 months with updates on our work done so far and our ongoing projects. Subscribe here now
Status of the Novartis study: Vibrant-HD
UPCOMING WEBINAR: Access to health care services and support for HD patients in Europe
Earlier this year we conducted an online survey trying to draw the European map of access to support and care for HD families and use this to advocate for good access for everyone affected by HD. We are proud to share that this survey was filled up by 800 HD family members and healthcare professionals from 31 countries.
The survey results will be presented in a Webinar by Filipa Júlio, on Thursday December 8 at 3 – 4 pm CET. After her presentation, we will open a Q&A and discussion session about what the results mean and how to best make use of them.
Thursday, December 8 2022
3 – 4pm CET
Moving Forward new section: The Bookshelf
The Moving Forward team is happy to announce a new feature in the Moving Forward webpage, the Bookshelf. The team has compiled many interesting books of different genres, all of them related to Huntington’s Disease (HD). Here you can find a comprehensive list with a summary of each book and additional information about where to buy or read it online
Since one of the main goals of Moving Forward is to increase the health literacy and provide reliable information to the HD community, the project team thought it would be interesting to have a new section dedicated to all you book lovers out there.
Meet our new EHA Board member - Rob Haselberg
Coping with Symptoms of Huntington's Disease
Our first day at the Huntington Study Group Meeting in Tampa, Florida
The 29th annual meeting of the Huntington Study Group started today with two exciting clinical trial round-ups. We learnt about the clinical trial updates from PTC Therapeutics, Roche, UniQure, Wave, Annexon, Neurocrine Biosciences, Prilenia, and Sage Therapeutics.
UniQure resume the patient enrollment at the higher dose for AMT-130 clinical trial
The story of Oda Felicia and ‘The Huntington Chorea project’
We are delighted to introduce Oda Felicia Abdelmaguid to the community. She is a Norwegian young woman who created her own personal project called “The Huntington Chorea Project“. This project is a collection of feelings in whatever shapeOda felt them during these years when the sorrow became a friend and the only thing steady in her sudden world of insecurity.
WEBINAR on the 'PTC518' clinical trial updates
The European Huntington Association, in collaboration with PTC Therapeutics, organized a Webinar on the progress of their Phase 2 clinical trial which has just started here in Europe.
The trial and treatment approach was presented by professor Sarah Tabrizi from University College London. She explained to us everything about the compound ‘PTC518’. After the presentation, Brian Beers, Global Project Leader and Amy-Lee Bredlau, Sr Dir Clinical Development from PTC, took part in the discussion.
Meeting with the ERN-RND 2022
The network partners met in Tuebingen in Germany at the end of September for the annual meeting. Since the previous face-to-face meeting the network has got a lot of new partnering clinics. From the initial 31, there is now 68 clinics from 24 countries. 48 of the clinics follow-up HD patients.
Poster exhibition - Some insights from the patient Organization projects
EHDN 2022 I Update on clinical trials
This year the EHDN 2022 Plenary Meeting was in Bologna, and we had the opportunity to go in person and learn about the latest updates on the current clinical trials.
Earlier in the year we were all hit by the suspended dosing in the VIBRANT- HD trial of Novartis. However, this year’s conference has demonstrated again that the hard work continues and there is some exciting progress that was presented in some of plenary sessions at the conference.
EHDN 2022 I How does the HD community live an International Conference?
Next EHA Conference in Belgium 2023!
On behalf of the EHA Board, we kindly invite you to join us on our next adventure in Blankenberge, Belgium.
The program for the upcoming EHA Conference will be open to HD family members and health care professionals.
Good news from Wave! Press Release (20th September)
The Select-HD is showing good achievements. 8 people have received the drug and the results shown so far is that after 85 days their levels of mutant huntingtin is reduced with 22 %. And this lowering is happening after just one injection of the drug.
🙌 This is encouraging and gives Wave good grounds to adjust and continue the trial! Select-HD is particularly interesting because the treatment only goes for the mutant Huntingtin and seems to leave the healthy Huntingtin untouched.
Prilenia Phase 3 Trial with Pridopidine – Why is it Different?
For all those who have been following Huntington’s disease research for some time, Pridopidine is an “old acquaintance”.
To date, several studies have been conducted with this drug, looking closely to its effect on dopamine. Although they have helped us to confirm the safety and good tolerability of pridopidine, none of these trials has been completely successful.
Wave Life Sciences announces a new active site in Italy for the 'Select HD' clinical trial
The study is a phase 1/2 and will look at the safety and how participants tolerate a new antisense oligonucleotide (ASO) in early-stage disease, with a drug called ‘WVE-003’.
The new facility that is recruiting is at the Centro Ricerche Cliniche Di Verona, and the principal investigator is Tomasso Bovi, MD.
PTC Therapeutics: 'PTC518' Phase 2 Clinical Trial updates
After these hard weeks for the Huntington’s Disease Community, we start September with renewed strength and hope!
The European Huntington Association in collaboration with PTC Therapeutics, announces an Online Webinar on the progress of their Phase 2 clinical trial which is just about starting up here in Europe.
The trial and treatment approach will be presented by professor Sarah Tabrizi from University College London. She will tell us more about the compound ‘PTC518’, a small molecule developed to reduce the production of mutated huntingtin (mHtt) As most of you know mHtt leads to the injury and death of neurons, which again results in the disease progression. After Tabrizi’s presentation there will be time for questions and comments and discussion. For this part of the webinar Brian Beers, Global Project Leader and Amy-Lee Bredlau, Sr Dir Clinical Development from PTC will be participating.
We hope you will use the opportunity to learn about this exciting trial and approach to treat Huntington’s Disease!
Novartis community letter: Dosing suspended in VIBRANT-HD trial of branaplam
Another set-back for HD research was announced by Novartis on August 8. The Vibrant-HD trial has stopped dosing due to the fact that some of the trial participants have exeperienced negative effects in the nerve system outside the brain.
Novartis has provided a community letter regarding Vibrant HD clinical trial updates.
Online talk with Dimitri Poffé - Thursday 14 July at 19:00 CET
Participate in the Photo Exhibition at the EHDN Plenary Meeting!
We need your photos and creativity this year!
Anybody interested in the topic – patients, family members, HD professionals, family friends and the general public – is invited to submit their photos illustrating the topic of “Abandonment: From Landscapes to Mindscapes”. You can submit more than one image (maximum of 2) if you wish.
EHDN2022 PLENARY MEETING
September 16th – 18th, 2022 in Bologna
Topics presented during the EHDN2022 Plenary Meeting will contain reports on recent advances in HD research and care. Aside from participating in the plenary and other parallel sessions, attendees will also have the opportunity to meet experts in the field of HD studies in more informal settings.
Adoption of the Global Action Plan on Epilepsy and other Neurological Disorders
We are delighted to support today’s announcement of the Global Action Plan on #Epilepsy & Neurological Disorders at the World Health Assembly.
The European Federation of Neurological Associations – EFNA, along with members of the OneNeurology partnership which EFNA co-founded, celebrates a major milestone for all those affected by neurological disorders today, with the adoption of the Intersectoral Global Action Plan on Epilepsy and Other Neurological Disorders (GAP) at this week’s 75th World Health Assembly.
The voice of the Huntington's Disease community
EHDN2022 PLENARY MEETING: September 16th – 18th, 2022 in Bologna
Topics presented during the EHDN2022 Plenary Meeting will contain reports on recent advances in HD research (in both basic and clinical sciences). Aside from participating in the plenary and other parallel sessions, attendees will also have the opportunity to meet experts in the field of HD studies in more informal settings.
In addition, the program also includes evening activities, ranging from informal meetings with industry experts to podium talks, movie night and a “Networking evening”.
Participate in the Moving Forward Forum!
- This is your moment to speak and be heard.
- This is your moment to learn from others and benefit from their experience.
- This is the moment to use your voice!
Video of Prilenia's Webinar "All you need to know about PROOF-HD”
If you missed the Webinar “All you need to know about PROOF-HD” hosted by Prilenia on May 4th, here is the full video.
May Awareness Month 2022 started with some updates from Michael Hayden, Andrew Feigin, Ralf Reilman and Anne Rosser about the Phase 3 trial where the investigative drug pridopidine is evaluated on HD patients. Close to 500 participants in North America and Europe are given either the active drug or placebo for more than one year.
The recruitment went fast and smooth and we can expect to have the first results published sometime first half of 2023. The speakers told us about the background and mechanisms of Pridopidine, status for the trial and potential next steps.
HELP US MAP HD PATIENTS AND FAMILIES ACCESS TO SUPPORT AND CARE 🤝
We know that many HD families and patients are struggling to get the care and support they need. But we don’t know where the gaps are. How is this for you? Do you get what you need and is it easy to access?
🌍 The European Huntington Association (EHA) has been working on an online survey trying to draw the European map of access to support and care for HD families and use this to advocate for good access for everyone affected by HD
It will take you about 10 minutes to respond. ✍️ We have the survey available in 20 languages.
Acting out disease: How patient Organizations Shaped Modern Medicine
On Thursday 21 April at 4pm, Swedish historian Ylva Söderfeldt will give a free (online) lecture on how patient organisations have contributed to the development of modern medicine.
The lecture will take place in English, but a Dutch simultaneous interpretation will be offered. More information about the lecture
HD Trial Finder - Watch the new video presentation of the new Webpage updates
EHA Board meeting in Bolognia, April 3rd 2022
The EHA is devasted by the Russian attack on Ukraine and all suffering this brings to people
The HD community is known for it’s warm and inclusive heart and we want the Ukrainians to know we are here and can help guide them to get in touch with experts if they suffer from Huntington’s Disease, says Astri Arnesen President of EHA. The HD associations in Europe will do their best to help you if you contact them here.
The Polish association has already helped a few Ukrainian HD patients fleeing the war and the President Danuta Lis is ready to help more.
Share your experience of seeking a diagnosis for your rare disease!
Take 20 minutes to complete the new #RareBarometer survey on the journey to diagnosis for people living with a rare disease!
How long did it take for your rare disease to be diagnosed? Are you still seeking a diagnosis for your rare disease?This survey is open to people living with a rare disease and their family members from any country in the world.
UniQure announced the completion of patient enrollment in the first two cohorts of its Clinical trial AMT-130 for HD
HD-CAB Training session 💻 in European Medicines Agency Processes for Marketing Authorisation and Patient Involvement
Assesing diagnosis and care pathways of people living with neurological disorders in Europe
Despite the higher prevalence among women of many neurological disorders (e.g. MS, migraine, Alzheimer’s) gender stereotypes can have a negative impact, leading to underdiagnosis and delayed treatment.
Take EFNA’s survey and help to identify the challenges facing all affected, either as patients or caregivers, from diagnosis to care pathway.
Huntington's disease is everywhere, in every country and region worldwide 🌍
Today is Rare Disease Day 2022! 🙌
As a part of this big community, we are trying to raise awareness and generate change for the 300 million people worldwide living with a rare disease, their families and carers 🤝
💜 And we also have our own community, the Huntington Disease community, in which we take care of each other. “We bring different strengths, but we are connected to the same stem”.
We also wanted to thank you for all your support and participation during this week. We collected all your incredible 📷 photos during this week and we are going to create a video sharing that Huntington Disease is everywhere, in every country 🌎
Let's raise some awareness for our community!
This year we need you more than ever! we want to raise awareness for Huntington Disease through social media, by using our filter for stories in Instagram or Facebook.
Here you can find the links for both social networks filter options that you can easily use and share:
Only 7 days away from Rare Disease Day 2022! 🙌
We want you to participate and share a picture of you showing that Huntington’s is everywhere. If you don’t want to appear in the picture you can also share a picture expressing your true colours , with a beautiful landscape, a flower or a monument itself.
You only need to post the picture, tag us (@EuroHuntington) and add the city where you took the picture.
Welcome to our basic online training on Huntington's disease
The Swedish Huntington Association has thanks to the funding from Allmänna Arvsfonden in Sweden been able to produce an online training course about Huntington’s Disease (HD) and how to provide best care. HD is a complex disease with a great variety of symptoms and this training resource will help you understand more about how to meet the needs of the patients throughout the course of the disease.
First European HD patients has been dosed with Uniqure’s gene therapy
First week of February another exciting step in HD research was taken, as the first two European patients were given Uniqure’s gene therapy in the ongoing Phase I/II trial. The drug is named AMT-130. Uniqure started the trial in the United States last year and is happy to be able to dose the first European participants as it has taken some time to identify the right participants and prepare the brain surgery this treatment requires.
JOIN-HD - Juvenile onset HD Global Registry
Online Education for professional care givers
Roche Webinar (24th January) recording available
After several months digging into all the information collected in the Generation HD1 trial Roche is ready tell us about their findings in a webinar organized by EHA in collaboration with Roche and EHDN. Lauren Boak, PhD and Peter McColgan, MD, PhD from Roche will present the key findings from the Generation HD1 trial and the Tominersen program.
Prof Anne Rosser, chair of EHDN, is participating in the following Q&A session and will help us understand the value of the findings and how we can make use of them for future work.
Vibrant HD clinical trial dosed first participant in January 2022!
Novartis managed to stick to the planned initiation of the Phase 2 trial and dosed the first participant just as we entered into 2022. To learn more about the project, Tina Leggett, on behalf of the European Huntington Association, travelled to the Novartis Campus in Switzerland. She interviewed Dimitri Papanicolaou, Jang Ho-Cha and Beth Borowsky, to learn more about Vibrant HD, a clinical trial Novartis is conducting where they test a drug named Branaplam.
New Webinar February 1st 2022, 3-4pm CET
Educational webinars on rare neurological, neuromuscular and movement disorders jointly organized by the European Reference Networks for Rare Neurological Diseases (ERN-RND) and Neuromuscular Diseases (ERN-EuroNMD) and the European Academy of Neurology (EAN). The speaker works as a research physician and PhD-candidate at the Neurology and Human Genetics department of the Leiden University Medical Center.
A new dawn for Tominersen
Some mornings are better than others and Tuesday January 18th was definitely one of the really good ones as I woke up to a beautiful sunrise and the news that Roche has decided to do another Phase II trial with Tominersen.
WEBINAR: Generation HD1 - what does the data tell us?
After several months digging into all the information collected in the Generation HD1 trial Roche is ready tell us about their findings in a webinar organized by EHA in collaboration with Roche and EHDN.
Online presenation on the Roche tominersen programme
This online meeting, hosted by EHDN, is geared towards a professional audience.
The meeting will include key findings from the Phase III GENERATION HD1 trial, followed by Q&A, then a panel discussion with recognised experts in the field of HD. The meeting will last approximately 90 minutes.
Happy New Year 2022
A new dawn for neurological diseases on the horizon
Our voice on behalf of people with neurological diseases (ND) has been heard! EU has decided to include ND on the list of health care priorities. This paves the way for a better future also for Huntington patients.
LET US TALK! Online course "Communication Skills for Healthcare Professionals"
For the first time in Russia, there will be an international online course for healthcare professionals organized by the European Huntington Association (EHA) – Moving Forward Project. Top experts from different countries will meet on one online platform to share their knowledge and experience in treating and caring for people with a rare genetic neurological disease – Huntington’s disease (HD).
The 3rd ERN-RND Winter School in collaboration with EPNS is dedicated to rehabilitation issues in patients with symptoms of a rare neurological disease
The first Moving Forward face-to-face meeting after Covid!
The Healthe-rnd consortium was finally able to meet again face-to-face in Prague.
Most of the partners were able to attend despite the ongoing restrictions due to the pandemic. The ones who couldn’t make it to Prague attended on Zoom. It was great to see each other again and also meet new faces as there have been additional people brought into work in the project.
Take the Survey before December 15th!
Moving Forward starts its activity in Spain, and we need you! We have created an online survey to find out the needs, concerns and desires of the Spanish community regarding their participation in research.
HD-CAB meeting with PTC
Moving Forward in Russia and Spain
HD-CAB new video
HD-CAB Website announcement!
The European Huntington Association (EHA), the International Huntington Association (IHA) and the Huntington’s Disease Youth Organisation (HDYO) are excited to officially announce the new website for this project, called HD-CAB. As a coalition of partners, their main mission is to represent the voice of the global HD community and provide HD community experience to regulators, industry, researchers and governing bodies.
You visit the new site here
Parkinson’s and Huntington’s Clinical Trials - the Participant Experience
EHA Board Meeting and General Assembly
MDS-ES and EHDN Joint Online Course Series
The EHDN in collaboration with the International Parkinson and Movement Disorder Society is delighted to announce a virtual HD course series ➡ Huntington’s Disease: From Foundational Principles to Assessment and Treatment
🗣 The primary purpose of this program is to provide information on foundational principles, assessment and management of Huntington’s disease.
💻 Online Course
📆 During October 2021 (3 Friday slots- 8, 15 & 22 October)
☑️ Registration: Free
Article about EHDN Remote Conference
After attending the EHDN Conference of 9-11th September 2021, Astri Arnesen wrote an article that summarize all the new updates on the HD Clinical Trials. This are some encouraging news!
FIFTY PERCENT - A Short Documentary
This incredible film explores the tension around Lillian, the daughter of an HD positive mum, deciding whether to get tested (to find out if she also carries the HD mutation) or not. This is something particularly important for the new generations, given the incredible advances that have been made since 1993.
The documentary has been released this week and is available to watch for free here
KICK-OFF MEETING ONLINE FOR HD-CAB
The Team Members of HD-CAB (Community Advisory Board) had last Saturday 4th their first globally Webinar with different advocates and partners.
How “emotional brain” is affected early in HD
Our results demonstrate that parts of the brain that manage emotions, the so-called limbic system, is affected early in HD mutation carriers. These changes may contribute to the development of psychiatric and cognitive symptoms. In many cases these are the most troublesome symptoms for both patients and their close family.
Our findings show that it’s not only the nerve cells that are affected in HD, but also other kinds of cells, like oligodendrocytes which play a major role in facilitating the communication between different parts in the brain, explains principal investigator Åsa Petersèn.
EHA has written an article for the Journal of Personalized Medicine
Survey for people with chronic diseases
As part of the European research consortium, IDEA-FAST is planning to conduct a large observational study with digital technology. The study is looking to develop new digital measures for fatigue and sleep disturbances for people with chronic diseases.Considering the importance of including patient voices in study designs a few minutes of your time would be highly appreciated.
To this aim, they would like to hear your opinion on how you think clinical studies using digital technologies should look like and to make sure the study is engaging and not too burdensome for participants with chronic diseases.
Multidisciplinary Treatment and Care WG meeting
Marleen van Walsem, Astri Arnesen and Ruth Veenhuizen, the lead facilitators of this working group, invite you to join this Zoom meeting.
Please feel welcome if you are proud of your multidisciplinary teamwork or in case you dream of improving multidisciplinary collaboration for HD families. In this meeting, we want to get to know each other and learn from each other on treatment, support and care for HD families.
Moving Forward Announcement!
The European Huntington Association is happy to announce the launch of the international project «Moving Forward» in Russia! The first city to present the project was Tomsk, which is situated in Syberia.
17 members of the HD families and several doctors-neurologists attended the interregional “School of Health”, organized by the center “Orphan People”.
HD-CAB, OneVoice4HD Announcement
The European Huntington Association (EHA), the International Huntington Association (IHA) and the Huntington Disease Youth Organisation (HDYO) are excited to officially announce their new project, called HD-CAB. As a coalition of partners, their main mission is to represent the voice of the global HD community and provide HD community experience to regulators, industry, researchers and governing bodies.
EFNA e-learning modules
The courses are video-based and divided into sections that can be watched independently at times that suit the viewer. Each module includes supporting notes and worksheets, as well as a the option to take a quiz on the content and receive a personalised certificate of completion.
EURORDIS High-level political forum 2021
Organised on the margins of the HLPF, the event is co-hosted by Spain, Qatar and Brazil – the Core Group of Member States promoting the call for a UN Resolution on Addressing the Challenges of Persons Living with a Rare Disease and their Families – together with the NGO Committee for Rare Diseases, Rare Diseases International and EURORDIS.
PROOF-HD reached 214 participants
PROOF-HD is a Phase 3, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of pridopidine in patients with early stage of Huntington Disease.
The recruitment still ongoing and the final number of participants needed is 480. You can find more information about this study and other ongoing trials in Europe on HD Trial Finder
This HD clinical trial will explore the safety, tolerability, and efficacy signals in 26 total patients with early manifest HD. “We are very pleased with the progress that we continue to make in this Phase 1-2 clinical trial and that we are now administering AMT-130 at the higher dose,” said Ricardo Dolmetsch, Ph.D., president of research at uniQure.
Scientists identify precisely how pridopidine works in models of Huntington’s disease.
Pridopidine is a drug developed to treat Huntington’s disease (HD) and now scientists have a clearer understanding of how it works in the body and brain.
Improving access to care and treatment for Huntington’s Disease patients and families
Today, only a minority of people affected by HD get the support and help they need. But there are solutions to this problem – expertise and knowledge exists – we only need to provide better access to all patients.
Life with HD comes in many flavours - alice wexler
During the webinar Annette
Listen to what the well known HD family member, academic and patient advocate from the US, Alice Wechsler, has to say about what life with HD can be like.
Join our Light
it up 4 HD!
How to take action:
- Light your house with purple or blue
- Take a picture
- Upload it on social media and add #LIGHTITUP4HD
- Tag us! @TheEuroHuntington
As you all remember in March the independent Data Monitoring Committee advised Roche to halt dosing of the drug Tominersen. On April 27 Scott Schobel, Medical Director & Clinical Science Leader at Roche shared the interim analyses of the results from the Generation HD1 at the CHDI therapeutics conference.
During the Awareness month we are organizing several webinars, articles and multiple ways to include everyone into our activities. Try our Facebook Frame for the May month!