Rezultati pretraživanja
  1. 8. ožu 2019.

    Wow !!! A single Cas9 guide RNA could generate > 7 megabases deletion -> on-target effect (FISH probe) in HEK293 cell line by P53 dependent mechanism on CEP disease !!! This is prevented with Cas9 nickase

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  2. 13. kol 2019.

    We’re proud to announce a disease education and promotional agreement with to raise awareness of acute hepatic (AHP). Learn more.

  3. 3. velj

    CHMP issues positive opinion for 's acute hepatic treatment , setting up EU approval in the next couple of months

  4. 13. tra 2019.

    Gene silencing as life changing treatment for patients. After 10 years of pain, this lady no longer takes painkillers as the treatment actually reverses the disease!

  5. Because I can't go out in the sunlight I'm a hat-wearing, gloves-wearing, little-skin-showing dork in real life.

  6. 20. stu 2019.

    FDA approves Givlaari (givosiran) for the treatment of adult patients with acute hepatic

  7. Doctors have used a new type of medicine called "gene silencing" to reverse a disease that leaves people in crippling pain. Exciting news

  8. I have new pieces of armor. My husband bought me these sun protective gloves with sleeves so I don’t fry as bad when I’m in the car or (god forbid!) outside. We will see how it go’s. I had a coolibar hoodie that I burned through. Cross fingers!

  9. 13. tra 2019.

    Acute Intermittent causes recurrent painful abdominal pain crises. Hard to diagnose unless one thinks of it. Thankfully rare but common topic in MRCP exams & more so now with gene-silencing treatment.

  10. 13. tra 2019.

    Great to see this article hit top 5 in BBC’s top read! is believed to have played a role in British history with George III and Mary Queen of Scots.

  11. 21. stu 2019.

    APF is pleased to announce that Givlaari (Givosiran) received approval for treatment of . "This RNAi treatment can prevent intolerable, painful attacks in patients w/ acute . It is a game changer." -Kristen Wheeden

  12. 5. ruj 2019.

    September is Pain Awareness Month . Pain can be silent, but you don’t have to be. For those suffering from acute intermittent , severe abdominal pain can last for hours or days. Learn more here:

  13. 10. ruj 2019.

    Today Oscar has given a impressive talk about the new advances in the Congenital Eritropoyetic therapy at conference, Milan. Joined efford coming from , PTS and .

  14. prije 14 sati
  15. 3. velj

    Well done to the team for securing a positive opinion from the for the treatment of the acute hepatic which once authorised by the EMA will address unmeet patients needs.

  16. 31. sij

    — التبرع لتكلفة علاج حنون على GoFundMe " أو عن طريق حساب أخوها على بنك مسقط : Account Number: 0378008596230015 (name): Nasser Albusaidi — ، ، . ,

    Ovo je potencijalno osjetljiv multimedijski sadržaj. Saznajte više
  17. 31. sij

    We’re pleased to announce that we’ve received a positive opinion from the EMA () Committee for Medicinal Products for Human Use () for our investigational therapeutic for the treatment of the acute hepatic .

  18. 30. sij
  19. 30. sij

    I am a Porphyria Warrior ...thank you for bringing awareness to 💜💜💜💜💜💜💜 made me love this show even more!!!

  20. 29. sij

    Wow this episode of brought life to the disease I was diagnosed in 1995 I am a 💜💜💜💜💜💜 "Thank You"

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