Rare disease research, treatment development, approvals, and care-access issues.
On July 1, 2026, the FDA expanded Casgevy (exagamglogene autotemcel) so some children with sickle cell disease (SCD) as young as age 2 can be evaluated for this one-time, CRISPR-based gene therapy. Here’s what changed, what the trial endpoints measured, what families can expect during treatment, and what safety and long-term follow-up questions still matter.
FDA approved TREGZI on June 30, 2026 to improve 1-year chronic GVHD–free survival after matched-donor allogeneic stem cell transplant in eligible adults. Here’s what the trial showed—and what remains uncertain.
On July 1, 2026, the FDA expanded Casgevy (exagamglogene autotemcel) so some children age 2 and older with sickle cell disease (SCD) can be evaluated for the gene-edited, stem-cell therapy—previously approved for older children. Here’s what the FDA change does and does not mean, what benefits were seen in pediatric data, and what risks and long-term monitoring families should discuss.
FDA’s July 1, 2026 expanded Casgevy (exa-cel) use for children as young as age 2 with severe sickle cell disease (SCD) and recurrent vaso-occlusive crises (VOCs). “Approved” doesn’t automatically mean every 3-year-old can receive it right away—eligibility details and specialized-center capacity for intensive monitoring matter.
With the first FDA-approved treatment for chronic hepatitis D now available, people who already have hepatitis B may want to ask about hepatitis D (HDV) testing. Here’s who to consider, what the drug is approved for, and what still isn’t proven.
The FDA has approved the first U.S. treatment for chronic hepatitis D. For people living with hepatitis B, that makes this a practical time to ask whether hepatitis D testing was ever done.
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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