November 22, 2019
Addressing the Barriers to Accessing Experimental Therapies
Read More
November 18, 2019
FDA Grants Anavex Rare Pediatric Disease Designation
Read More
November 18, 2019
EMA Grants Orchard Accelerated Assessment of Therapy for Patients with MLD
Read More
October 31, 2019
FDA Places Partial Hold on Trials of Zolgensma Injected into Spinal Fluid
Read More
October 14, 2019
The Mother of Invention Strikes Again
Read More
October 8, 2019
FDA Awards 12 Grants to Fund Rare Disease Therapy Clinical Trials
Read More
October 8, 2019
FDA Provides $4.1 Million for Two Natural History Studies
Read More
September 23, 2019
FDA Places Hold on Abeona Ahead of Pivotal Trial of Cell Therapy for RDEB
Read More
September 6, 2019
FDA Clarifies Accelerated Pathway for Medical Devices for Rare Disease Indications
Read More
September 6, 2019
Takeda Issues Recall for Natpara
Read More
August 29, 2019
Report of Patient Deaths Creates Questions about Vertex’s CF Drug
Read More
August 13, 2019
Senate Pressure Grows on Novartis over Gene Therapy Data Manipulation
Read More
Load More
X