Related Experiment Video
Updated: Jul 10, 2026

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
Published on: September 5, 2016
271st ENMC international workshop: Towards a unifying effort to fight Kennedy's disease. 20-22 October 2023,
M Pennuto1, P F Pradat2, G Sorarù3
1Department of Biomedical Sciences (DBS), University of Padova, 35131 Padova, Italy; Veneto Institute of Molecular Medicine (VIMM), Padova 35100, Italy.
Abstract:
The workshop held in the Netherlands from October 20-22, 2023, united 27 scientists from academia, healthcare, and industry representing 11 countries, alongside four patient and charity representatives. Focused on Kennedy's Disease (KD), also known as spinal and bulbar muscular atrophy (SBMA), the workshop aimed to consolidate knowledge, align on clinical trial designs, and promote participative medicine for effective treatments. Discussions emphasized KD's molecular mechanisms, highlighting its status as a neuromuscular disorder with motor neuron degeneration. Strategies for therapeutic intervention, including AR activity modulation and targeting post-translational modifications, were proposed. The need for diagnostic, prognostic, and target engagement biomarkers was stressed. Challenges in patient stratification and clinical trial recruitment were acknowledged, with the International KD/SBMA Registry praised for its role. The workshop concluded with a patient-focused session, underscoring challenges in KD diagnosis and the vital support provided by patient associations.
Related Concept Videos
Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
International Nursing Organizations I
ICN member organizations work to advance the field of nursing and healthcare via policies, partnerships, lobbying, professional...
International Nursing Organizations II
The WHO provides expert team support, including funding, vaccines, testing, and treatment tools at the country level to fight...
Treatment Resistent Cancers
Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
Huntington Disease l: Introduction

