Clinical trials devoted to rare diseases represent crucial initiatives aimed at addressing the distinctive challenges encountered by individuals affected by uncommon medical conditions. Research conducted in this domain entails navigating restricted patient populations, often geographically dispersed, necessitating specialized approaches for the design and execution of trials. The formulation of clinical trials for rare diseases demands meticulous consideration of disease-specific factors, encompassing natural history, diagnostic criteria, and the identification of appropriate outcome measures. Collaborative efforts involving researchers, healthcare professionals, and patient advocacy groups play a pivotal role in facilitating patient recruitment and ensuring the practicality of trial implementation.
In the realm of rare disease clinical trials, exploration of innovative therapies, including gene therapies and targeted treatments, is common, reflecting the imperative for pioneering solutions in the absence of established standard-of-care options. Regulatory agencies increasingly acknowledge the significance of cultivating an environment conducive to rare disease research, streamlining approval processes for orphan drugs. The success of clinical trials in rare diseases hinges on patient engagement and advocacy, underscoring the importance of adopting a patient-centric approach. The dynamic landscape of precision medicine further underscores the need to tailor interventions to the specific genetic and molecular characteristics of rare diseases, instilling hope for enhanced outcomes and treatments for individuals grappling with these complex medical conditions.
Title : The impact of metal-decorated polymeric nanodots on proton relaxivity
Paulo Cesar De Morais, Catholic University of Brasilia, Brazil
Title : Hepatotoxic botanicals-shadows of pearls
Consolato M Sergi, Universities of Alberta and Ottawa, Canada
Title : Exploring classical ayurvedic drugs in hypertension
Prashant Bhokardankar, Datta Meghe Ayurved College, India
Title : Principles and standards for managing healthcare transformation towards personalized, preventive, predictive, participative precision medicine ecosystems
Bernd Blobel, University of Regensburg, Germany
Title : Personalized and Precision Medicine (PPM) as a unique healthcare model based on design-inspired biotech- & biopharma-driven applications to secure the human healthcare and biosafety
Sergey Suchkov, N.D. Zelinskii Institute for Organic Chemistry of the Russian Academy of Sciences & InMedStar, Russian Federation
Title : A unique role and and impact of catalytic antibodies (abzymes) in clinical practice: A novel strategy for predicting and preventing relapse in chronic autoimmune conditions
Sergey Suchkov, N.D. Zelinskii Institute for Organic Chemistry of the Russian Academy of Sciences & InMedStar, Russian Federation
Title : The promise of nanotechnology in personalized & precision medicine: Drug discovery & development being partnered with nanotechnologies via the revolution at the nanoscale
Sergey Suchkov, N.D. Zelinskii Institute for Organic Chemistry of the Russian Academy of Sciences & InMedStar, Russian Federation
Title : The promising future of the unique translational tool to manage beta-cell population renewal and regeneration to secure the post-diabetic period
Sergey Suchkov, N.D. Zelinskii Institute for Organic Chemistry of the Russian Academy of Sciences & InMedStar, Russian Federation
Title : Easily injectable, organic solvent free self assembled hydrogel platform for endoscope mediated gastrointestinal polypectomy
Hitasha Vithalani , IIT Gandhinagar, India