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 "targeted treatment"

ResearchPUBMEDMar 26

[New ways in interdisciplinarity: internal medicine meets human genetics : When frequent conditions become rare and rare conditions become frequent].

Doctors are learning that common diseases are actually made up of many different rare subtypes when scientists look at their genetic makeup. By understanding the specific genetic changes in each person's disease, doctors can create targeted treatments that work better. This article uses chronic myeloid leukemia as an example—a treatment that targets one specific genetic change has turned a deadly disease into one people can live with for decades.

WHY IT MATTERSIf you have been diagnosed with a common cancer or disease, genetic testing may reveal you have a rare molecular subtype that qualifies you for a precision medicine treatment not available to patients with other genetic variations of the same disease.
💬 Ask your doctorchronic myeloid leukemiacancer
PolicyPUBMEDMar 26

Medicine Development and Access for Rare Diseases: Can We Do Better?

Scientists and doctors are saying that the system for developing and selling medicines for rare diseases needs improvement. New discoveries in genetics have helped create targeted treatments, but these medicines are often very expensive and hard to access. The article calls on researchers, doctors, and other groups to work together to make rare disease medicines more affordable and available to patients who need them.

WHY IT MATTERSIf you or a loved one has a rare disease, this research highlights why medicines for your condition may be expensive or hard to get — and pushes for systemic changes that could improve your access to treatment options.
ℹ Good to knowrare diseases (general)inherited metabolic diseases
Clinical trialCLINICALTRIALSMar 26

Trial Results Posted: Testing AZD9291 as Potentially Targeted Treatment in Cancers With EGFR Genetic Changes (MATCH-Subprotocol E) (NCT06303167)

Researchers tested a cancer drug called osimertinib (AZD9291) in 19 patients with advanced cancers that have specific genetic changes in a gene called EGFR. The drug works by blocking a mutated protein that helps cancer cells grow. This trial is now complete and results have been posted.

WHY IT MATTERSThis trial provides evidence that osimertinib may work against cancers with EGFR mutations across different cancer types, potentially offering a treatment option for patients whose tumors have this specific genetic change.
💬 Ask your doctorAdvanced LymphomaAdvanced Malignant Solid NeoplasmRefractory Lymphoma

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