Gene Therapy for Duchenne Muscular Dystrophy: Cash’s Story

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  • Опубліковано 30 жов 2023
  • Children’s Hospital of Philadelphia now offers the first-ever gene therapy treatment for Duchenne Muscular Dystrophy (DMD) to patients like 6-year-old Cash. This breakthrough treatment slows the progression of symptoms and can improve strength and endurance for patients with DMD.
  • Наука та технологія

КОМЕНТАРІ • 40

  • @chukwudiobinna3079
    @chukwudiobinna3079 7 місяців тому +1

    Great hope. Well done breakthrough makers

  • @petersampson5202
    @petersampson5202 7 місяців тому +4

    DEAR CASH & EVERYONE AT CHILDREN'S HOSPITAL OF PHILADELPHIA,
    GOD BLESS YOU MY FRIENDS. I AM VERY PROUD OF YOU AND VERY HAPPY FOR YOU. I HOPE ALL OF YOUR DREAMS COME TRUE. 🙏
    I LOVE YOU SO VERY MUCH. 🙏
    GOD BLESS YOU SO VERY MUCH CASH. 🙏
    I LOVE CHILDREN'S HOSPITAL OF PHILADELPHIA SO VERY MUCH. 🙏
    SINCERELY PETER

    • @elmehdiamine9103
      @elmehdiamine9103 3 місяці тому

      My son 6 years was the same hospital with the same doctor he hase dmd too I hope God help all the kids and parent too

    • @petersampson5202
      @petersampson5202 3 місяці тому +1

      @@elmehdiamine9103 GOD BLESS YOUR SON. GOD BLESS ALL OF THE BEAUTIFUL DOCTORS. GOD BLESS ALL OF THE BEAUTIFUL PARENTS. GOD BLESS EVERYONE AT CHILDREN'S HOSPITAL OF PHILADELPHIA. I LOVE THE CHILDREN'S HOSPITAL OF PHILADELPHIA SO VERY MUCH. I WILL PRAY FOR EVERYONE. 🙏

    • @sarabnagra3456
      @sarabnagra3456 10 днів тому

      How much does this cost?

  • @rituchoudhary1699
    @rituchoudhary1699 8 днів тому

    Is there gene therapy for kcnq2 gene varients ??

  • @HanifKhan-ui7ht
    @HanifKhan-ui7ht 4 місяці тому

    Iam from Guyana my grandson is 10 years old he have dmd he is not walking we need help for him

  • @user-pu1si4mv4l
    @user-pu1si4mv4l 19 днів тому

    My brothers son ( 13 years old ) having this but we dont have treatment in Sri Lanka.we need help and guide from youll.

    • @ChildrensHospPhila
      @ChildrensHospPhila  19 днів тому

      Please contact our Global Patient Services program to explore treatment options at Children’s Hospital of Philadelphia. You can call 1-267-426-6298 or visit ms.spr.ly/6052YXeXr for additional information.

  • @rajarani479
    @rajarani479 3 місяці тому +1

    my son is 9 years old he is DMD patient (7 exon is deletion) in genetic report im in pakistan please help me 😢

  • @natashaayudan5791
    @natashaayudan5791 3 місяці тому

    I wish older children could get it I would give anything to stop my sons rate of progression I hate this disease so much

  • @user-vx5np7qz7r
    @user-vx5np7qz7r 3 місяці тому

    My son is ten year old with DMD I am from Mauritius .He stop walking since last year please help if there is any treatment let me know.

    • @ChildrensHospPhila
      @ChildrensHospPhila  3 місяці тому

      Hello. Please contact our Global Patient Services program to explore treatment options at Children’s Hospital of Philadelphia. You can call 1-267-426-6298 or visit ms.spr.ly/6052cRRYm for additional information.

  • @yangweichen-rp9gx
    @yangweichen-rp9gx 3 місяці тому

    I am from China. My son is DMD patient. He is 5 years old. Is it possible to cure? And how much would be the cost?❤

    • @ChildrensHospPhila
      @ChildrensHospPhila  3 місяці тому +1

      Hello. Please contact our Global Patient Services program to explore treatment options at Children’s Hospital of Philadelphia. You can call 1-267-426-6298 or visit ms.spr.ly/6052cRRYm for additional information.

    • @yangweichen-rp9gx
      @yangweichen-rp9gx 3 місяці тому

      @@ChildrensHospPhila Thanks for your feedback.I will check it out

  • @LGRacing123
    @LGRacing123 Місяць тому

    Im from Srilanka .My son also suffering from DMD.now he's 8years old.still he is working slowly .please please help me if there is any possibility .thank tou .

    • @ChildrensHospPhila
      @ChildrensHospPhila  Місяць тому

      Please contact our Global Patient Services program to explore treatment options at Children’s Hospital of Philadelphia. You can call 1-267-426-6298 or visit ms.spr.ly/6050Y6icC for additional information.

  • @sarabnagra3456
    @sarabnagra3456 11 днів тому

    My sister's son in DMD exons 48-52 deletion.He is 2 year's 11 months old. please hlp me what is suitable treatment for him. I am form Punjab

    • @ChildrensHospPhila
      @ChildrensHospPhila  11 днів тому

      Please contact our Global Patient Services program to explore treatment options at Children’s Hospital of Philadelphia. You can call 1-267-426-6298 or visit ms.spr.ly/6051YbU41 for additional information.

    • @rituchoudhary1699
      @rituchoudhary1699 8 днів тому

      Did you get any treatment for your sister's son ? Plz tell if any

  • @jaysimoes3705
    @jaysimoes3705 3 місяці тому

    Yes great and all. I want to see results, of course I can find some results but I would like to see much more results and since what I have seen (have been shown?) are results within months we should be able to see these in more kids. Not just a few.

  • @Rashvanth2021
    @Rashvanth2021 4 місяці тому

    My son 2years 11month old affect in DMD (14 exon is deletion or duplication) in gentic report. Please help me what is suitable treatment for my baby ( i am in India) please..... share me

    • @devikabai309
      @devikabai309 4 місяці тому

      Hi madam for Duchenne Muscular Dystrophy and every type of Dystrophy can give treatment in Nimhans Hospital in Bangalore

    • @ChildrensHospPhila
      @ChildrensHospPhila  4 місяці тому

      Please contact our Global Patient Services program to explore treatment options at Children’s Hospital of Philadelphia. You can call 1-267-426-6298 or visit ms.spr.ly/6052iFuJk for additional information.

    • @jaysimoes3705
      @jaysimoes3705 3 місяці тому

      Exon 14 can be tricky, so inform yourself. I think 1-9 and 17 so 14 should in theory be fine but again ask your specialist.

    • @Rashvanth2021
      @Rashvanth2021 3 місяці тому

      14 exons (Exon 30 -43) deletion or duplication in genetic report

  • @ramsita8198
    @ramsita8198 5 місяців тому +1

    Wow good news

  • @DavidCandy-iu7sr
    @DavidCandy-iu7sr 2 місяці тому

    Successful Hope

  • @samaymeena5001
    @samaymeena5001 2 місяці тому

    Please help my son is suffering ducchne muscular dystrophy since 8 years please help 😭😭 I am from India

    • @ChildrensHospPhila
      @ChildrensHospPhila  2 місяці тому

      Hello. Please contact our Global Patient Services program to explore treatment options at Children’s Hospital of Philadelphia. You can call 1-267-426-6298 or visit ms.spr.ly/6052cRRYm for additional information.

  • @EdrianDometita-zb7wm
    @EdrianDometita-zb7wm 2 місяці тому

    Hi I'm edrian from Philippines my brother suffere DMD since 7yr.old please help my brother

    • @ChildrensHospPhila
      @ChildrensHospPhila  2 місяці тому

      Please contact our Global Patient Services program to explore treatment options at Children’s Hospital of Philadelphia. You can call 1-267-426-6298 or visit ms.spr.ly/6050ctwEo for additional information.

  • @bgmi_Moses
    @bgmi_Moses 3 місяці тому +1

    I'm from India am DMD patient I'm 21years old help me to cure 😢

    • @ChildrensHospPhila
      @ChildrensHospPhila  3 місяці тому

      Hi! Please contact our Global Patient Services program to explore treatment options at Children’s Hospital of Philadelphia. You can call 1-267-426-6298 or visit ms.spr.ly/6052cRRYm for additional information.

    • @balwantrajput4264
      @balwantrajput4264 2 місяці тому

      Hello . Can I get your contact no.

    • @ChildrensHospPhila
      @ChildrensHospPhila  2 місяці тому

      To explore treatment options at Children's Hospital of Philadelphia, please contact us at 1-800-879-2467 or visit our website ms.spr.ly/6054cmH3m.

    • @pickledbumblebeeviolinist
      @pickledbumblebeeviolinist 2 місяці тому

      Our sons have Duchenne. They are 10 and 13. Any help or hope for them?

    • @ChildrensHospPhila
      @ChildrensHospPhila  2 місяці тому

      Thank you for contacting us. To explore treatment options at Children's Hospital of Philadelphia, please contact us at 1-800-879-2467 or visit our website ms.spr.ly/6056cQTaa.