CMT Research Foundation
CMT Research Foundation
  • Видео 97
  • Просмотров 20 486

Видео

Clinical Development of CLC-1 Inhibitors For the Treatment of CMT
Просмотров 139 часов назад
Martin Skov, PhD, Innovation Manager, NMD Pharma
You and the Future of Clinical Trials in CMT
Просмотров 149 часов назад
Dottie Caplan, Senior Vice President of Patient Advocacy and Engagement, Applied Therapeutics
A Guide to Clinical Research: One Family's Journey
Просмотров 199 часов назад
Jenneen DiFiore, Executive Director of Neurology, PPD, a Division of Thermo Fisher Scientific
Fueling Progress With ENDGAME - The Campaign to End CMT1A
Просмотров 219 часов назад
Susan Ruediger, Co-Founder and Chief Mission Officer, CMTRF Peter de Silva, Board Chair, CMTRF Riann Egusquiza, PhD, Director of Research, CMTRF
Lessons Learned From SOD1 ALS Clinical Development: Opportunities for CMT Drug Development
Просмотров 119 часов назад
Toby Ferguson, MD, PhD, Chief Medical Officer, Voyager Therapeutics
The State of Drug Development for CMT
Просмотров 139 часов назад
Robert Prior, PhD, Postdoctoral Researcher, University of Bonn, Germany
Living With CMT
Просмотров 209 часов назад
Marjorie Ritchie, CMT Patient
What is Happening to my Nerves and Body
Просмотров 109 часов назад
Bruce Carter, PhD, Professor of Biochemistry, Vanderbilt University
Living With CMT
Просмотров 59 часов назад
Alexandre Hoyau, President, CMT France
UNSTOPPABLE
Просмотров 89 часов назад
Jamal Hill, US Paralympian Medalist, CMT Patient
Opening/Patients Taking Action
Просмотров 69 часов назад
Patient Day Opening Cleary Simpson, CMTRF CEO Peter de Silva, CMTRF Board Chair
Patient Voices: How Your Scientific Progress Provides Hope
Просмотров 129 часов назад
Adrienne Weinstein, Endgame Campaign Committee Member, CMT Research Foundation
Patient Voices - Living With CMT
Просмотров 249 часов назад
Eloise Schlafly, CMT advocate and CMT patient discusses her journey living with CMT
TRPV4 Inhibition in the Treatment of CMT2C
Просмотров 709 часов назад
David Goldstein, PhD, Co-Founder and CEO of Actio Biosciences
Supporting Translation of CMT1A Gene Therapy Through AAV-mediated Silencing of PMP22
Просмотров 169 часов назад
Supporting Translation of CMT1A Gene Therapy Through AAV-mediated Silencing of PMP22
Development of Gene Replacement Therapy for CMT4A Demyelinating Neuropathy
Просмотров 189 часов назад
Development of Gene Replacement Therapy for CMT4A Demyelinating Neuropathy
The Development of Tissue Tropic AAVs - Considerations Inside Out
Просмотров 129 часов назад
The Development of Tissue Tropic AAVs - Considerations Inside Out
Developing Lipid Nanoparticles for Non-Liver RNA Delivery
Просмотров 189 часов назад
Developing Lipid Nanoparticles for Non-Liver RNA Delivery
Current Status of Clinical Outcome Assessments and Biomarkers in CMT
Просмотров 209 часов назад
Current Status of Clinical Outcome Assessments and Biomarkers in CMT
Understanding the Criteria For Pursuing a Drug Program for CMT
Просмотров 239 часов назад
Understanding the Criteria For Pursuing a Drug Program for CMT
Discovery of Small Molecules that Alter the Trafficking of PMP22 as A Potential Route
Просмотров 139 часов назад
Discovery of Small Molecules that Alter the Trafficking of PMP22 as A Potential Route
Developing Best-in-class HDAC6 Inhibitors for the Treatment of CMT1A
Просмотров 89 часов назад
Developing Best-in-class HDAC6 Inhibitors for the Treatment of CMT1A
FDA Efforts to Advance Medical Product Development for Rare Diseases
Просмотров 129 часов назад
FDA Efforts to Advance Medical Product Development for Rare Diseases
Govorestat for CMT-SORD: Designing a Clinical Trial for a Slow Progressing CMT
Просмотров 139 часов назад
Govorestat for CMT-SORD: Designing a Clinical Trial for a Slow Progressing CMT
Understanding Basic Biological Mechanisms to Identify Therapeutic Targets in CMT
Просмотров 89 часов назад
Understanding Basic Biological Mechanisms to Identify Therapeutic Targets in CMT
Clinical Development of CLC- Inhibitor for the Treatment of Charcot-Marie-Tooth Disease
Просмотров 169 часов назад
Clinical Development of CLC- Inhibitor for the Treatment of Charcot-Marie-Tooth Disease
Beyond The Diagnosis: Patient Stories, Expert Insights. Episode 3
Просмотров 7414 дней назад
Beyond The Diagnosis: Patient Stories, Expert Insights. Episode 3
Beyond The Diagnosis: Patient Stories, Expert Insights. Episode 2
Просмотров 132Месяц назад
Beyond The Diagnosis: Patient Stories, Expert Insights. Episode 2
Beyond The Diagnosis: Patient Stories, Expert Insights. Episode 1
Просмотров 1662 месяца назад
Beyond The Diagnosis: Patient Stories, Expert Insights. Episode 1

Комментарии

  • @aashrinavlog
    @aashrinavlog 2 дня назад

    I have CMT. I am 42 years old .I am not any treatments and diagnosis yet.

  • @aashrinavlog
    @aashrinavlog 2 дня назад

    I have CMT 1A I am from Nepal 🇳🇵🇳🇵

  • @simonjohnson2272
    @simonjohnson2272 2 дня назад

    I Have CMT1A , From New Zealand

  • @gangaramjatrajolakallan149
    @gangaramjatrajolakallan149 Месяц назад

    I am 24 yr male from india i suffer from last 8 yr this disease (cmt) , gene therapy is possible for treat it then i want treatment . What is the cost of gene therapy for cmt

  • @MilanGaži-y7y
    @MilanGaži-y7y Месяц назад

    Hello i from Slovakia and have a question for u my aunt have 14 years and doctors diagnoze him CMT4G type RUSSE at 6 years thay make the generic tests . My question is if this medication Can help him . And second question is how the illnes will continue i want to know if it is posible he Can end Up on wheelchair . Thanks for every information .

  • @AB1Vampire
    @AB1Vampire 2 месяца назад

    No mention of CMT4? Great description of how the transcription process takes RNA to Protein status.

  • @twistnbend4jewlz
    @twistnbend4jewlz 2 месяца назад

    I wish these trials were offered in Canada

  • @idakingsnorth1190
    @idakingsnorth1190 2 месяца назад

    You walk so much better than I and you don’t have AFOS. I’m 65 and I’m finding it more challenging this last year .

  • @MdAlif-j7l
    @MdAlif-j7l 3 месяца назад

    How much time it will take I am I am hearing it from the age of 15 and now I am 21 can you please tell me ?

    • @cmtresearchfoundation
      @cmtresearchfoundation 2 месяца назад

      Determining how long a drug development program will take to become an approved drug is challenging because each program is unique and it's unpredictable if a drug will meet the necessary milestones for approval. CMTRF currently has 5 CMT projects that have resulted in drug candidates that are now moving towards clinical trials and we aim to continue to fund more projects to increase the chances of getting approved treatments for CMT. If you would like to stay updated on the progress of these projects, please visit cmtrf.org/ and sign up for our email mailing list.

  • @MdAlif-j7l
    @MdAlif-j7l 3 месяца назад

    Dear, I am 21 year aged I am hearing form the age of 15 please do something because I can see how my life is ruin after few year I will be unable to walk.

  • @ShuvamDadal
    @ShuvamDadal 3 месяца назад

    can gene therapy stop the progression of cmt?😂

    • @cmtresearchfoundation
      @cmtresearchfoundation 2 месяца назад

      Genetic medicines have the potential to stop or even reverse the progression of diseases because they target the underlying genetic cause. This makes them promising therapeutic approaches to explore for CMT. As of today, CMTRF has funded 11 projects that explore various genetic technologies for CMT. To date, these projects have resulted in 3 genetic-based drug candidates that are now moving forward towards human clinical trials. For more information on genetic medicines and to stay up to date on all the CMT genetic-based therapy projects we fund, please visit cmtrf.org/gene-therapy/

  • @vinitthakur6168
    @vinitthakur6168 4 месяца назад

    What a Inspiring story ❤ , Thanks u Peter uncle One day I also hope that I will also contribute to this association 😊

  • @sivaprathipati7783
    @sivaprathipati7783 4 месяца назад

    I am from India my son is suffering with CMT1A, age present 7 yeas. when can i expect durg for CMT please let me know.

  • @michaelglenn5459
    @michaelglenn5459 4 месяца назад

    Peter, a very Inspiring and Courageous story! I didn't have any idea about your CMT. But your video has certainly increased my respect for you and my awareness of CMT. Blessings & Best Wishes!

  • @pr1ce213
    @pr1ce213 4 месяца назад

    Beautifully made

  • @gelilatesfaye2341
    @gelilatesfaye2341 4 месяца назад

    I have Cmt1e too. Stay strong queen

  • @ShuvamDadal
    @ShuvamDadal 5 месяцев назад

    Many people are suffer by this disease.

  • @gamaltaher9714
    @gamaltaher9714 6 месяцев назад

    Thanks

  • @Amine.81
    @Amine.81 7 месяцев назад

    Thank you so much for answering my question regarding my 9 years old daughter. I'm glad to get in touch with you. Appreciate your cooperation

  • @gamaltaher9714
    @gamaltaher9714 8 месяцев назад

    Thanks

  • @gamaltaher9714
    @gamaltaher9714 8 месяцев назад

    Thanks

  • @gamaltaher9714
    @gamaltaher9714 8 месяцев назад

    Thanks

  • @DrDenham
    @DrDenham 9 месяцев назад

    I am a physician with CMT2A. I am 47. I have lost a lot of strength in the last two years. What type of results could individuals with advanced symptoms expect to see once treatments are available (i.e. will our muscles be able to regrow, will it just stop further atrophy, will we wear be able to stop wearing braces?) There is little talk of what is possible after treatments are available.

  • @Amine.81
    @Amine.81 10 месяцев назад

    Wow, what a nice motivation 👏 🎉

  • @Amine.81
    @Amine.81 10 месяцев назад

    Proud of your efforts 👌 Please keep going. You are changing peoples lives ❤

  • @Sunshine-jk9jq
    @Sunshine-jk9jq 11 месяцев назад

    I have learned more from your video than any CMT or MD charity in the UK. Well done. My symptoms manifested at 6 months of age and now at 69 I am facing using a wheelchair permanently and no known treatment to help in decades!!

  • @Fintan23754
    @Fintan23754 Год назад

    Many thanks for sharing 👍I hope to make it to the 2024 Convention.

  • @Amine.81
    @Amine.81 Год назад

    Thank you for your effort.. Appreciated.

  • @Amine.81
    @Amine.81 Год назад

    I am looking forward to watch the meeting videos of September 22 23 2023. All our support keep 🙏 going ❤

  • @God_is_my_savior-316
    @God_is_my_savior-316 Год назад

    Amitriptyline 150 mg,3600 MG gabapentine,8mg ramelteon helps my cmt severe neuropathic pain in my feet.

  • @lainieh4928
    @lainieh4928 Год назад

    Thank you

  • @simonjohnson2272
    @simonjohnson2272 Год назад

    Hi Lily, Well done to you for being able to talk about this, My name is Simon Johnson aged 50 that also have CMT so I fully understand what you are going though and about to . The hardest thing for me is people not understanding , But I had to find a happy place which I have just only found. I have a wife and 3 amazing Kids and a Grandson so you can still can enjoy life . one of the best things for myself was reaching to the CMT community about a year or 2 ago . Every day just keep smiling and keep doing what you are doing because you are helping people in what you are doing, you have helped me today just listening to your Conversation with Susan and thank also to Susan. lots of love from New Zealand

  • @Donnamg100
    @Donnamg100 2 года назад

    When will clinical trials begin in USA?

    • @cmtresearchfoundation
      @cmtresearchfoundation 2 года назад

      Hello! Please follow along our research updates at cmtrf.org/research-we-fund and subscribe to our newsletter to always be informed cmtrf.org/subscribe

  • @Hesam0000
    @Hesam0000 2 года назад

    Ofcourse the NIH Clinic of Maryland has already cured or found a cure for genetic disease like CMT by repairing the bad Chromosome in the Gene.

  • @God_is_my_savior-316
    @God_is_my_savior-316 2 года назад

    Severe severe burning pain!

  • @God_is_my_savior-316
    @God_is_my_savior-316 2 года назад

    My feet feel like they are on fire 🔥 I am considering amputating my feet to alleviate the 10/10 torturous severe pain!

  • @God_is_my_savior-316
    @God_is_my_savior-316 2 года назад

    I have cmt1a. The feet burning agonizing pain at night is 10000 worse than the weakness.

  • @ramazan9857
    @ramazan9857 2 года назад

    When will remyelination therapy be released?

    • @cmtresearchfoundation
      @cmtresearchfoundation 2 года назад

      Hello! Please follow along our research updates at cmtrf.org/research-we-fund and subscribe to our newsletter to always be informed cmtrf.org/subscribe

  • @bluefatherj
    @bluefatherj 2 года назад

    This is great news, so sad this video only has 40 views.

    • @cmtresearchfoundation
      @cmtresearchfoundation 2 года назад

      We are glad to share the news with you. Please share amongst your circle and social to help bring awareness. Thank you for watching!

  • @rebelinaction2260
    @rebelinaction2260 2 года назад

    Hi there, how did you set up the Town Hall with Dr Peter Marks? I am active within my community and have received some queries here that I can’t answer with authority. Please can somebody assist me in setting one up? I think my community would greatly benefit from hearing from Dr Marks answer queries directly!

    • @cmtresearchfoundation
      @cmtresearchfoundation 2 года назад

      Hello! This was a video call with Dr. Marks and the CMTRF, not a town hall. Unfortunately, we cannot help with scheduling a meeting with Dr. Marks. If you have questions about CMT, please feel free to email us at info@cmtrf.org

  • @Sunshine-jk9jq
    @Sunshine-jk9jq 3 года назад

    Excellent video. I have had advanced CMT 1a since birth and after 66 years this video helps me to understand yet more about my condition.

  • @russelllash8044
    @russelllash8044 4 года назад

    Thank you. Lets find a cure. I'm so tired of hurting.

  • @brianparker5777
    @brianparker5777 4 года назад

    0:26 dating-near.online

  • @laurensteers6344
    @laurensteers6344 5 лет назад

    amazing progress i 12 months

  • @michaelsutton6072
    @michaelsutton6072 5 лет назад

    Amazing work, CMTRF

  • @oliverdebattista4812
    @oliverdebattista4812 5 лет назад

    A big Thank you