Erica Marini recaps October’s neurology pharmacy news, from FDA updates and new multiple sclerosis trial data to a fatal epilepsy case that shows the danger of patients relying on AI for medication advice.

Experts highlighted how Diamond-Blackfan anemia illustrates the scientific, financial, and manufacturing challenges that can prevent promising gene therapies for ultra-rare diseases from reaching patients.

Without head-to-head trials, clinical data, payer requirements, and clinician comfort determine first-line regimen selection, while markers such as KEAP1 and STK11 prompt questions about adding a CTLA-4 inhibitor.

Pembrolizumab and cemiplimab dominate frontline practice, yet approval thresholds, histology-specific backbones, and formulations keep clinicians from treating PD-1 agents as fully interchangeable in the absence of head-to-head data.

Experts highlighted recent gene therapy approvals for inherited immune disorders while addressing ongoing challenges in manufacturing, long-term safety, and patient access.

Expected in the first half of 2028, the planned separation would create a stand-alone retail pharmacy company with nearly 30,000 pharmacists.