Clinical Development Disease Presentation 
Drug Target Review
APRIL 7, 2025
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. 2 This progress has implications for diagnosis, therapeutic efficacy, and potentially establishing clinically relevant endpoints.
FDA Law Blog: Drug Discovery
NOVEMBER 10, 2024
Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
Drug Target Review
JULY 30, 2025
Patient recruitment: CGT trials face unique recruitment and retention challenges, particularly with rare disease populations. CGT trials face unique recruitment and retention challenges, particularly with rare disease populations. Modern IRT platforms address these through advanced patient management features and integrations.
The Pharma Data
JUNE 12, 2025
The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.
Drug Target Review
JULY 1, 2025
Clinical trials are expensive, slow and often limited by outdated design constraints. Placebo arms, in particular, create ethical and logistical hurdles, especially in areas like rare disease and oncology. They offer patient-specific outcome predictions, generated using machine learning models trained on real historical clinical data.
The Pharma Data
JUNE 6, 2025
At this premier global meeting focused on thrombosis, hemostasis, and bleeding disorders, Sanofi will present compelling data across 18 scientific abstracts, including five oral presentations. In addition, two poster presentations will offer deeper insights into the LUNA 3 trial.
The Pharma Data
JUNE 11, 2025
Dapirolizumab Pegol Demonstrates Promise in Phase 3 SLE Trial, Showing Improvements in Fatigue and Disease Activity at EULAR 2025 UCB (Euronext Brussels: UCB) and Biogen Inc. The latest data from the PHOENYCS GO trial reinforce the potential of dapirolizumab pegol to improve disease outcomes in people with moderate-to-severe SLE.
Expert insights. Personalized for you.
Let's personalize your content