by CheckRare Staff | Aug 26, 2026
FDA approval is a major milestone, but for patients with rare diseases, it is only the beginning. In this episode of The CheckRare Brief, we look at two new rare disease approvals, ongoing drug supply problems affecting patients, and a major acquisition that...
by CheckRare Staff | Aug 20, 2026
On this week’s episode of The CheckRare Brief, we discuss FDA’s approval of Zenbexus (iberdomide) to treat patients with multiple myeloma, the complete response letter issued to ITM-11 to treat patients with neuroendocrine tumors, and safety concerns about...
by CheckRare Staff | Aug 18, 2026
Rare endocrine disorders can be difficult to recognize, challenging to diagnose, and even harder for patients to navigate. For many patients, the journey to an accurate diagnosis can take years. Rare Endocrine Exchange, a new podcast from CheckRare, aims to change...
by CheckRare Staff | Aug 13, 2026
On this week’s episode of The CheckRare Brief, we discuss FDA’s approval of Orzeyful (oveporexton) to treat patients with narcolepsy type 1, CAMP4’s first-in-human clinical trial for patients with SYNGAP1-related disorders, and BioMarin’s termination for...
by CheckRare Staff | Aug 1, 2026
August is SMA Awareness Month, a time dedicated to increasing understanding of spinal muscular atrophy (SMA) and supporting the individuals and families impacted by this rare genetic neuromuscular condition. SMA affects the motor neurons, causing progressive muscle...
