Rare disease research, treatment development, approvals, and care-access issues.
FDA has approved the first gene therapy for a rare OTOF-related hearing loss, but the eligible group is small and the evidence is still early. Here is what U.S. families should know about testing, treatment, and follow-up.
Three FDA decisions in March 2026 gave families in the rare-disease world something unusual: approved options for conditions that previously had few or none. Here’s what changed, what the evidence looked like, and what still needs to be watched closely.
The FDA says it wants to make room for individualized treatments in ultra-rare diseases without lowering the bar for evidence. A new draft framework, plus several 2026 approvals, shows how regulators are trying to balance flexibility with proof that a therapy targets the disease’s root cause.
A new FDA-approved treatment for Menkes disease raises the stakes for early diagnosis. The hard part now is whether newborn screening systems can find ultra-rare disorders fast enough, accurately enough, and fairly enough to help babies in time.
The FDA has approved the first treatment for children with Menkes disease, a rare and often severe genetic disorder that starts in infancy. The bigger story is how quickly the disease can cause harm — and why faster diagnosis may matter as much as treatment itself.
The CDC’s 2026 Adult Immunization Schedule includes specific guidance for adults who are immunocompromised, including many people living with lupus. Here’s what it means for influenza, COVID-19, pneumococcal, RSV, and shingles vaccines — and how medication timing and insurance coverage come into play.
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