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CIFFREO Study Fails Primary End Point, Gene Therapy for Duchenne Shows Promise, Deflazacort Generic Approved by FDA
16 Jun 2024 11:25 GMT
… ), Pfizer’s investigational mini-dystrophin gene therapy, did not meet its primary … program. In the trial, the gene therapy failed to achieve the primary … -associated virus (AAV8) vector-based gene therapy, was safe and well tolerated …
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Gene therapy offers hope for patients with hearing loss
16 Jun 2024 01:13 GMT
… or genetic causes of deafness. Gene therapy typically involves replacing or supplementing … non-viral methods are revolutionising gene therapy for genetic hearing loss. Lipid … the re-administration of the gene therapy vector, since the primary administration …
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Experts Debate Scope of Access to Gene Therapy for Patients With Hemophilia
15 Jun 2024 16:52 GMT
… on any association between gene therapy and malignancy.
Gene therapy carries a risk … A after valoctocogene roxaparvovec gene therapy—a nontrivial proportion.1
… Indirect treatment comparisons of the gene therapy etranacogene dezaparvovec versus extended half …
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What New Gene Therapy Means For Sickle Cell Patients
15 Jun 2024 15:38 GMT
… to begin commercially approved gene therapy that could potentially cure … complications.
The promise of gene therapy provides hope for curing … to the CDC.
Although gene therapy addresses the root cause … insurance will cover his gene therapy, it remains to be …
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Gene therapy shows encouraging results in sickle cell patients
15 Jun 2024 12:41 GMT
San Francisco, Jun 15 (IANS): Scientists have reported an innovative gene-based treatment that modifies a patient’s own blood-forming stem cells to correct the mutation responsible for sickle cell disease.
The clinical trial aimed at discovering a cure for …
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BIO CEO’s patriotic pivot; Step forward for Lilly’s Alzheimer’s drug; Pfizer’s gene therapy setback; Foresite’s $900M fund
15 Jun 2024 10:06 GMT
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Listen: Duchenne gene therapy setback, Alzheimer’s drug endorsement & why a STAT reporter buys weed (for journalism)
14 Jun 2024 09:07 GMT
… chat about Pfizer’s Duchenne gene therapy setback, the likely approval of … the failure of Pfizer’s gene therapy study, go here, and a …
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STAT+: Europe is set to shake up the way it assesses new medicines. Gene therapy makers are worried
14 Jun 2024 09:08 GMT
LONDON — A major shakeup in how the benefits of medicines are assessed in Europe is looming, and developers of treatments like gene therapies are already arguing that what they see as flaws in the new system could entrench the issues they’ve had getting …
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SMARCB1 Gene Therapy Using a Novel Tumor-Targeted Nanomedicine Enhances Anti-Cancer Efficacy in a Mouse Model of Atypical Teratoid Rhabdoid Tumors
14 Jun 2024 07:40 GMT
… of patients with ATRT.
Discussion
Gene therapy, which involves replacement of a … . Transferrin-liposome-mediated systemic p53 gene therapy in combination with radiation results … CC, et al. Systemic p53 gene therapy of cancer with immunolipoplexes targeted …
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Emergence of Cell and Gene Therapy Community in Singapore
14 Jun 2024 06:37 GMT
… often necessitates the use of gene therapy techniques to transform the … a bona fide Cell and Gene Therapy (CGT) community with more … like the Cell and Gene Therapy Catapult and the Advanced … biologics and Cell and Gene therapy companies and their key …