by Peter Ciszewski | Aug 8, 2026
The CheckRare Brief delivers a concise summary of the week’s most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights, scientific publications, and industry news that matter to healthcare...
by Peter Ciszewski | Jan 1, 2026
Below is the list of important regulatory dates for all orphan drugs for 2026. Prescription Drug User Fee Act (PDUFA) dates refer to deadlines for the FDA to review new drugs. PDUFA DateOrphan DrugIndicationCompanyStatus 1.5.2026Cablivi (caplacizumab)Acquired...
by Peter Ciszewski | Dec 26, 2025
Current Status of Gene Therapies in Rare Neuromuscular Disorders Alan Beggs, PhD and Julie A Parsons, MD Drs. Beggs and Parsons discuss the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the...
by Peter Ciszewski | Dec 21, 2025
Current Status of Gene Therapy in Lysosomal Storage Disorders Nicola Longo MD, PhD and Mark Roberts, MD In this eight-part podcast series, Drs. Longo and Roberts discuss the current status of gene therapies in lysosomal storage disorders. This content is derived from...
by Peter Ciszewski | Dec 15, 2025
Catching the Clues, Changing the Course of Lysosomal Storage Disorders Professor Yoshikatsu Eto, Dr. Nicole Muschol, Professor Patrício Aguiar, Dr. Robert Hopkin This interactive symposium explores the patient journey across the lysosomal storage disorder (LSD)...
