Rezultati pretraživanja
  1. prije 10 minuta

    Meet Kohen, a Smith-Kingsmore Warrior, and learn how his family supports his development to overcome challenges!

  2. prije 29 minuta

    Meet Kin Ping. He has retinitis pigmentosa, a that includes symptoms like difficulty seeing at night & the gradual deterioration of sight. Check out his story & save the date for Feb 29

  3. prije 45 minuta

    CDH International is very grateful to UK actor, Charles Lawson, for this great 25th Anniversary shout out and challenge for the kids! (**language warning)

  4. prije 45 minuta

    CDH International is very grateful to UK actor, Charles Lawson, for this great 25th Anniversary shout out and challenge for the kids! (**language warning)

  5. prije 51 minutu

    Come and chat with the TREAT-NMD representatives at the 2020 Conference in Evry for information on how we can support the neuromuscular community

    2 korisnika označavaju da im se sviđa
  6. prije 51 minutu

    Receiving a diagnosis is difficult on many fronts. When Ella was diagnosed, her family found it difficult to get support because so few had heard of it. Check out their story, then take the .

    1 reply 2 proslijeđena tweeta 3 korisnika označavaju da im se sviđa
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  7. prije 1 sat
    Odgovor korisniku/ci

    We know. I have 3 CVID,Keinbock,Presiers & brittle bones. I depend on life saving monthly. I will die without them. Let me know so I can prepare to die.

  8. prije 1 sat

    We fully pledge our support to the patients and families of Rare Disease. We will continue to support and advocate for the implementation of the National Rare Disease Plan.

    - Signs & symptoms of 70% of rare diseases appear exclusively in childhood. At least 4% of children are diagnosed with a by age 17. are chronic, progressive, degenerative and often life-threatening.
  9. prije 2 sata

    . has entered into a multi-year drug discovery collaboration with CRO . The companies will launch multiple integrated programs across Takeda’s four core therapeutic areas: , , and

  10. prije 2 sata

    Thank U & for highlighting the As some1 w it, I know title will trigger some bc we have been dismissed medically & told 2 just think positive 2 get through pain- & we know it isnt that easy But mindset= big part of equation Be open 2 it

    Thanks to who ! Today they featured Amberly who developed after her leg was severely injured in an accident: “34 surgeries was a lot but it was really the easiest part. The hard part is getting through the pain every day.”
    1 reply 1 proslijeđeni tweet 1 korisnik označava da mu se sviđa
  11. prije 2 sata

    Dasatinib as a Treatment for Chronic Myeloid Leukemia

  12. prije 2 sata
    Odgovor korisniku/ci

    Thank you! Interesting fact I just found out in my research No chemotherapy drug for cats is approved If she is a candidate for treatment it will be off-label just like her mom 😀 They only have approved treatments for dogs

    1 reply
  13. prije 2 sata

    “Prepare for your daughter to die very soon,” the doctor told them.

  14. prije 2 sata

    We’re pleased to present an aromatic L-amino acid decarboxylase () deficiency learning session on 2/7 at the first Genetic Syndromes with Movement Disorders and Epilepsy symposium in Barcelona, Spain.

    1 proslijeđeni tweet 2 korisnika označavaju da im se sviđa
  15. prije 2 sata

    The ECD Global Alliance is excited to announce we have a new addition to the ECD Care Center Network! Welcome Dr. and the O’Neal Comprehensive Cancer Center at the University of Alabama at Birmingham (UAB)!

  16. prije 2 sata

    Missed the release? Check out our article on how IMPC researchers are using new categorisation systems to find new candidate genes associated with .

    1 proslijeđeni tweet 2 korisnika označavaju da im se sviđa
  17. prije 2 sata

    Psyched to start new career with Viral Vectors team! BD for components and services, including vectors, related to and gene-modified . .

    1 reply 2 korisnika označavaju da im se sviđa
  18. prije 2 sata

    My baby was diagnosed with She is only 6 & spent 5 of those years getting me through the hardest time in my life; my accident & journey Hardest part=this AM I woke up w her pouncing on my face wanting to play

    4 korisnika označavaju da im se sviđa
  19. prije 3 sata

    Answer: According to , to be classified as a , the condition must impact 200,000 people or less.

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  20. prije 3 sata

    This significant contribution by Viralgen will go a long way to provide access to AAV manufacturing in order accelerate programs for ultra-rare diseases which has been a significant bottleneck in gene therapy.

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