Company entered into an asset purchase agreement with Johnson & Johnson (J&J) to acquire all interests in botaretigene sparoparvovec (bota-vec) for the treatment of X-linked retinitis pigmentosa (XLRP ...
Clinically meaningful improvements in xerostomia symptoms measured by PRO Xerostomia Questionnaire (XQ) maintained out to 3 years post treatment ...
Roche is launching a Phase 3 trial of the controversial Duchenne gene therapy Elevidys, hoping it could lead to approval in ...
Pfizer announced in February 2025 that it was taking Beqvez (fidanacogene elaparvovec), its gene therapy for hemophilia B, ...
Pfizer and BioMarin have pulled their hemophilia gene therapies, citing low demand. To prepare for his presentation at the ...
In a time when what defines gender is being questioned and discussed, a new study reveals that single changes in DNA make dramatic differences.
Just imagine, if the person responsible for evaluating vaccines at the FDA was required to have experience in that field ...
Presentation to include 3-year data from all cohorts of the Phase 1 AQUAx Clinical Study- Cohort-level data and individual patient data from ...
At a first meeting on pediatric gene and cell therapy trials, participants discussed when enrolling children before adults is ethical and when to wait.
An experimental drug designed to silence a gene strongly linked to Parkinson's disease has shown encouraging effects in a ...
Peripheral nerve injuries, often caused by traumatic events such as car accidents, falls or battlefield injuries, can leave patients with long-term weakness, numbness or loss of function. Despite ...
By 85, according to the Social Security Administration, it’s more like 10 percent. Death comes quickly.) Soon after the carry-on incident, Scott saw an advert for an experimental gene therapy from a ...
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