Bringing Cell Therapies to Rare Disease Patients in Europe
Bringing Cell Therapies to Rare Disease Patients in Europe Rare diseases are defined by sheer numbers of new patients per population. This definition is not helpful for the affected patient and his/her family. These conditions often lack effective treatment options due to their low prevalence. In addition, there are challenges in developing traditional pharmaceuticals for […]
Hope for Rare Diseases: Drug Repurposing for Better Patient Care in Europe
Hope for Rare Diseases Drug Repurposing for Better Patient Care in Europe For patients with rare genetic diseases, the struggle extends far beyond the challenges of the disease itself. Multiple co-morbidities often accompany these conditions leading to complex polypharmacy treatment. The reliance on multiple medications exposes patients to a higher risk of adverse effects from […]
The Global Landscape of Rare Disease Treatments
The Global Landscape of Rare Disease Treatments Legal and Regulatory Considerations for Pharma and Biotech Companies Rare diseases, affecting a small percentage of the population, often face a lack of research funding and treatment options. However, the global pharmaceutical and biotechnology industry is increasingly focusing on developing therapies for these conditions. To incentivize this crucial […]
Navigating Gene Therapy Commercialization in Europe: A Q&A with Novartis’ Joshi Venugopal
Navigating Gene Therapy Commercialization in Europe A Q&A with Joshi Venugopal, General Manager of Gene Therapies and Rare Diseases, Europe Opinions expressed here are personal and not that of Novartis* Gene therapies hold immense promise for revolutionizing the treatment of rare diseases, offering potential cures where previously only symptom management was possible. However, the path […]
RARE DISEASE CLINICAL TRIALS: A Collaborative Approach to Design and Execution
Rare Disease Clinical Trials A Collaborative Approach to Design and Execution Patients who suffer from rare diseases need access to effective and safe medications. Given the low incidence of the diseases, the collaboration between regulatory authorities, producers, insurances, and market access teams is essential for pharmaceutical companies seeking to bring new medications to patients and […]
