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#clinicaltrials

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United States · AAV gene therapy for autosomal recessive deafness 9: a single-arm trial - United StatesGene therapy for congenital deafness has shown promising results in children but lacks data in older populations. We conducted a single-arm trial of adeno-associated virus (AAV)-OTOF gene therapy using the Anc80L65 capsid in ten participants with autosomal recessive deafness 9 aged 1.5 to 23.9 years at five sites in China. The primary endpoints were safety and tolerability within 5 years, and secondary endpoints assessed auditory function. Initial findings from the ten patients with 6–12 months of follow-up, including one patient who received two injections, revealed that the therapy was well tolerated, with 162 grade I/II adverse events. Decreased neutrophil percentage was the most common event (16 of 162). All ten participants had at least 6 months of follow-up and improved their pure-tone-average hearing level from baseline 106 ± 9 (mean ± s.d.) to 52 ± 30 decibels (dB). Other secondary endpoints showed similar improvements, including the average click auditory brainstem response (ABR) threshold, the tone-burst ABR threshold and the auditory steady-state response (101 ± 1 to 48 ± 26 dB, 91 ± 4 to 57 ± 19 dB and 80 ± 14 to 64 ± 21 dB, respectively). Post hoc analyses were conducted to evaluate the timecourse and factors contributing to the hearing improvement. Therapeutic effect was rapid, taking 1 month to achieve most of the overall hearing improvement. On an individual level, click and tone-burst ABR thresholds, but not the auditory steady-state response, reliably predicted the behavioral pure-tone-average thresholds after 4 months (R2 = 0.68, 0.73 and 0.17, respectively). An age-dependent therapeutic effect was observed, with optimal outcomes in 5- to 8-year-olds. These preliminary results show that AAV-OTOF was safe and well tolerated in patients ranging from toddlerhood to adulthood. The trial remains ongoing and requires extended follow-up to confirm the long-term safety and efficacy. ClinicalTrials.gov registration: NCT05901480 . Preliminary results from an investigator-initiated clinical trial showed that an AAV-OTOF gene therapy was safe and led to hearing improvements in ten patients with congenital deafness with 6–12 months of follow-up, including in a teenager and a young adult, expanding the age range from previous trials.

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Interministerial report on the importance of communicating the results of #clinicaltrials in France.
The report identifies the main levers for improvement and makes operational recommendations designed to support all stakeholders involved. The aim of these recommendations is to facilitate the effective implementation of the European regulation while also promoting a culture of transparency for the benefit of research, patients and society as a whole.
#openscience
ouvrirlascience.fr/publication

alojapan.com/1273118/japans-st Japan’s Stem Cell Scientists Claim Breakthrough in Parkinson’s Treatment #ClinicalTrials #dopamine #IpsCells #Japan #JapanNews #JunTakahashi #KyotoUniversity #NeurodegenerativeDiseases #neuroscience #news #Parkinson'sDisease #RegenerativeMedicine #STEMCELLS #SumitomoPharma In a world-first clinical trial, Japanese scientists have successfully implanted lab-grown brain cells into Parkinson’s disease patients — and early results suggest the tre…