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Several discussions reference the high success rate of 91% not experiencing major functional disabilities at 24 months and the additional context of long-term risks such as insertional oncogenesis. Phrases like 'exciting times' and 'hope for ALD patients' indicate a hopeful tone, while terms like 'caveats' and 'ongoing risk' show a cautious perspective as well. This mix of optimism and concern shapes the overall sentiment, demonstrating a deep engagement with the topic.
Most discussions express support for the promising results of the therapy, though some concerns about potential risks are noted.
There is a high level of curiosity surrounding the advancements in gene therapy, as many participants highlight its implications for treatment.
Numerous participants have provided detailed analyses and raised questions about the therapy's methodology and associated risks.
Opinions on the therapy's overall impact vary, with some emphasizing its significance while others remain cautious.
Very good commentary in @NEJM about the development of hematological malignancy in recipients of lentiviral gene therapy for cerebral adrenoleukodystrophy. Could it be that the promoter of this specific lentiviral vector was responsible or is it a more widespread issue? The https://t.co/LUBG6L4M71
Original Article: Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/EW3byXqKt7 Editorial: Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/tmH8GhNUei #Hematology https://t.co/CNhlvVEkwD
Original Article: Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/0Z9LN9weW5 Editorial: Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/tmH8GhNUei #Neurology https://t.co/PUOBAO889L
Thirty-two patients with cerebral adrenoleukodystrophy received lentiviral gene therapy, and after a median of 6 years most were free of major disability. Myelodysplastic syndrome developed in one patient. Full study results: https://t.co/0Z9LN9weW5 https://t.co/e6KnkREZk4
We live in amazing times. Gene Therapy for Adrenoleukodystrophy. https://t.co/Jja3aNmmqM
Important. 7 of 67 patients who had lentiviral gene therapy for cerebral adrenoleukodystrophy developed hematological cancers. 5 of these 7 underwent allogeneic bone marrow transplantation. Paper is behind a pay wall. https://t.co/dEvst9l6PE. Although for most #hemophilia https://t.co/l0KLIUovrU
Understanding hematologic cancer risks in Eli Cel treated CALD patients. This video discusses the promising results of elivaldogene autotemcel as a treatment for cerebral adrenoleukodystrophy and addresses concerns…
This week's news covers a range of scientific developments, including the promising gene therapy for cerebral adrenoleukodystrophy, which showed stability in neurological function over six years. Additionally, we…
@CarlSmytheCells @otrbikers @Alex_on_A14 @bepeco Exciting times ahead, obviously going to be bumps in the road, but we're in the 2nd or 3rd generation of vectors now. LNPs might be another option soon. Some of this stuff was sci-fi when I graduated. https://t.co/gmMhVUcJs3 https://t.co/QFR9Me18gc https://t.co/q2ExjlHFM9
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/8lCUxJ12Zm
Thoughts on this? >> Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy - nejm.org: Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy nejm.org >> Comment below! #strategy #competitiveintelligence #marketing #competitivemarketing #pharmaceutical #pharma #biotech #healthcare
CALD - cerebral adrenoleukodystrophy. There is nothing for these kids 1:17k $BLUE 91% free of major func disability at 24mon (n=32) 81% free at 6 yrs **NO insertional oncogenesis**
RT @ProfMakris: Very good commentary in @NEJM about the development of hematological malignancy in recipients of lentiviral gene therapy fo…
RT @MGH_RI: In a new study published in @NEJM, researchers from @MGHNeurology and colleagues found that a gene therapy shows long-term ben…
#Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy showed at a median follow-up of 6 years no major functional disabilities. However, #insertional oncogenesis is an ongoing risk associated with the integration of viral vectors. https://t.co/O3EqRUvDXb
RT @MGH_RI: In a new study published in @NEJM, researchers from @MGHNeurology and colleagues found that a gene therapy shows long-term ben…
RT @MGH_RI: In a new study published in @NEJM, researchers from @MGHNeurology and colleagues found that a gene therapy shows long-term ben…
RT @MGH_RI: In a new study published in @NEJM, researchers from @MGHNeurology and colleagues found that a gene therapy shows long-term ben…
In a new study published in @NEJM, researchers from @MGHNeurology and colleagues found that a gene therapy shows long-term benefit for patients with a rare pediatric brain disease. Read more: https://t.co/Skp5ogMfK4 https://t.co/GeYz331KD0 https://t.co/BXLhAENF7c
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy #science #publication #research #publications https://t.co/gMT1znTB1p
Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy #science #publication #research #publications https://t.co/En7m1iXBOk
RT @NEJM: Original Article: Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/0Z9LN9weW5 Editorial: Weighing the Ris…
RT @NEJM: Original Article: Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/0Z9LN9weW5 Editorial: Weighing the Ris…
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy | New England Journal of Medicine https://t.co/y0toeFdiDQ
RT @NEJM: Original Article: Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/0Z9LN9weW5 Editorial: Weighing the Ris…
Original Article: Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/0Z9LN9weW5 Editorial: Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/tmH8GhNUei #Neurology https://t.co/PUOBAO889L
RT @NEJM: Original Article: Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/EW3byXqKt7 Editorial: We…
RT @NEJM: Original Article: Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/EW3byXqKt7 Editorial: We…
RT @NEJM: Original Article: Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/EW3byXqKt7 Editorial: We…
RT @NEJM: Original Article: Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/EW3byXqKt7 Editorial: We…
RT @NEJM: Original Article: Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/EW3byXqKt7 Editorial: We…
RT @NEJM: Original Article: Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/EW3byXqKt7 Editorial: We…
Original Article: Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/EW3byXqKt7 Editorial: Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/tmH8GhNUei #Hematology https://t.co/CNhlvVEkwD
We live in amazing times. Gene Therapy for Adrenoleukodystrophy. https://t.co/Jja3aNmmqM
RT @ProfMakris: Very good commentary in @NEJM about the development of hematological malignancy in recipients of lentiviral gene therapy fo…
@JuanitaZap26975 @neuroblues7 https://t.co/gA01kI6WTZ
@dixieliano @neuroblues7 https://t.co/gA01kI6WTZ
@neuroblues7 Lentiviral…? https://t.co/gA01kI6WTZ
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy - https://t.co/Taprv6J1vx
RT @NEJM: Editorial: Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/tmH8GhNUei
RT @ProfMakris: Important. 7 of 67 patients who had lentiviral gene therapy for cerebral adrenoleukodystrophy developed hematological cance…
Hope for ALD patients- with caveats: Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy | New England Journal of Medicine https://t.co/PIq63zL43Y
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy | New England Journal of Medicine https://t.co/y0toeFdiDQ
あのマッドマックスで有名なジョージミラーが監督したことでも有名なロレンツォのオイルで扱われたAdrenoleukodystrophyに対する遺伝子治療の報告です 時代は進んでいます Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy | New England Journal of Medicine https://t.co/2ZKWrxhvN1
RT @ProfMakris: Very good commentary in @NEJM about the development of hematological malignancy in recipients of lentiviral gene therapy fo…
RT @ProfMakris: Very good commentary in @NEJM about the development of hematological malignancy in recipients of lentiviral gene therapy fo…
Very good commentary in @NEJM about the development of hematological malignancy in recipients of lentiviral gene therapy for cerebral adrenoleukodystrophy. Could it be that the promoter of this specific lentiviral vector was responsible or is it a more widespread issue? The https://t.co/LUBG6L4M71
Important. 7 of 67 patients who had lentiviral gene therapy for cerebral adrenoleukodystrophy developed hematological cancers. 5 of these 7 underwent allogeneic bone marrow transplantation. Paper is behind a pay wall. https://t.co/dEvst9l6PE. Although for most #hemophilia https://t.co/l0KLIUovrU
Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/yjgEbmjEfx https://t.co/1lYR96TgAd
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/KWiVDbXErf https://t.co/LsMUOqRnmQ
RT @NEJM: Thirty-two patients with cerebral adrenoleukodystrophy received lentiviral gene therapy, and after a median of 6 years most were…
RT @NEJM: Thirty-two patients with cerebral adrenoleukodystrophy received lentiviral gene therapy, and after a median of 6 years most were…
RT @NEJM: Editorial: Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/tmH8GhNUei
RT @NEJM: Thirty-two patients with cerebral adrenoleukodystrophy received lentiviral gene therapy, and after a median of 6 years most were…
RT @NEJM: Thirty-two patients with cerebral adrenoleukodystrophy received lentiviral gene therapy, and after a median of 6 years most were…
RT @NEJM: Editorial: Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/tmH8GhNUei
Editorial: Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/tmH8GhNUei
Thirty-two patients with cerebral adrenoleukodystrophy received lentiviral gene therapy, and after a median of 6 years most were free of major disability. Myelodysplastic syndrome developed in one patient. Full study results: https://t.co/0Z9LN9weW5 https://t.co/e6KnkREZk4
RT @NEJM: Editorial: Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/tmH8GhNUei
Editorial: Weighing the Risks of Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy https://t.co/tmH8GhNUei
NEJM also published updated study data from Skysona clinical trial program, and an editorial on its benefit-risk: https://t.co/sOjnXbchFN https://t.co/Kz5YCikqAD