Rare disease news

FDA approvals, research breakthroughs, clinical trials, and advocacy updates

Curated and summarized by AI for patients and caregivers

🔍
AllDrug approvalsClinical trialsResearchGrants & fundingAdvocacy & policyPipeline
Show:All newsBreaking onlyImportant & breaking
Date:7 days30 days90 daysAll time

3 articles from the last 90 days matching "pharmacokinetics"

Clinical trialUNITERARE5 days ago

New Recruiting Trial: A UGT1A1 Genotype-Directed Study of Belinostat Pharmacokinetics and Toxicity

Researchers are testing a cancer drug called belinostat to see how it works in people's bodies and what side effects it causes. The study is special because it looks at a genetic test (UGT1A1) that affects how people process this drug, so doctors can figure out the right dose for each person based on their genes.

WHY IT MATTERSThis trial is now recruiting patients and uses genetic testing to personalize belinostat dosing, which could help predict who will tolerate the drug better and reduce serious side effects.
👁 Watch this space
Clinical trialUNITERAREMay 4

New Recruiting Trial: Pharmacokinetics Of Emulsified Avacopan Applied By NG Tube

Researchers at Mayo Clinic are testing a new way to give a medicine called avacopan to patients who cannot swallow pills normally. Instead of swallowing, the medicine will be delivered through a tube that goes down the nose into the stomach. This Phase 1 trial is looking at how the body absorbs and processes this emulsified (liquid) form of the drug.

WHY IT MATTERSThis trial could help patients with conditions treated by avacopan who have difficulty swallowing or need alternative delivery methods access this medication more easily.
👁 Watch this space
Clinical trialUNITERAREApr 30

New Recruiting Trial: A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG)

Researchers are testing a new medicine called inebilizumab in children who have generalized myasthenia gravis (gMG), a rare condition where the immune system attacks muscles and causes weakness. This study will check how the medicine works in children's bodies, whether it's safe, and what side effects it might cause. The study is now accepting patients and is expected to start in April 2026.

WHY IT MATTERSThis is the first trial testing inebilizumab specifically in children with gMG—previous studies only included adults—so it could lead to the first approved treatment option for this age group.
→ You can act on thisgeneralized myasthenia gravis

Get personalized rare disease news

Follow your conditions to see news about the diseases that matter to you — FDA approvals, trial openings, and research breakthroughs.

Create free account →Browse diseases