News

Researchers investigate whether the location of a mutation in the PIK3CA gene affects the mutation’s ability to drive cancerous growth.

After a high-volume flu season driven by outbreak of the mutated H3N2 strain, Sanofi has bolstered its vaccine class with options specific to older populations.

Clinical trials consistently demonstrate that fecal microbiota treatment is an effective treatment strategy for recurrent CDI, yet standardization practices vary.

The second member of the FDA-approved thrombopoietin receptor agonist drug class was proven efficacious and safe in a pair of phase 3 trials.

Leading artificial intelligence companies have partnered up to develop new drug candidates for Duchenne muscular dystrophy.

The 2 assays from Ortho Clinical Diagnosis consolidates the company's package of devices and systems designed to give patients frequent diagnoses.

In a new study, researchers say they’ve improved on a method of delivering probiotics to premature newborns in the fight against deadly gut infections.

The optimal duration of dual antiplatelet therapy remains unclear, despite being proven efficacious for roughly 2 decades.

The FDA granted an orphan designation to Surface Oncology’s SRF231 for the treatment of multiple myeloma, a rare blood cancer.

The FDA has approved iobenguane I 131 (Azedra) injection for intravenous use for the treatment of adults and adolescents 12 years of age and older with rare adrenal gland tumors such as pheochromocytoma or paraganglioma that cannot be surgically removed, have spread beyond the original tumor site, and require systemic anticancer therapy.

Positive data collected from 2 phase 2 trials evaluating bardoxolone methyl in patients with chronic kidney disease (CKD) caused by Alport syndrome and autosomal dominant polycystic kidney disease (ADPKD) have been reported.

Researchers find that blocking one or more genes in the HSF1 pathway could represent a new approach in treating the rare, aggressive disease.

The FDA has granted a fast track designation to diazoxide choline controlled-release (DCCR) for the treatment of Prader-Willi syndrome.

The device supplants Abbott's own 10-day Libre system as the longest-lasting self-applied continuous glucose monitoring system on the US market.

Patients who received electronic asthma control plans had fewer asthma exacerbations, but more research is needed to find out how much of an impact such interventions have over time.

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

A new analysis suggests the benefits of universal HCV screening would outweigh the significant costs.

The FDA accepted the NDA for NKTR-181, a drug with slowed entry into the brain that attenuates the dopamine release underlying euphoria.

Three-quarters of patients with aspirin-exacerbated respiratory disease often have allergies to alcohol. A new study finds a common therapy for the one can also help the other.

Risk of scar development increased from three-fold at 2 years to 4.5-fold at 5 years, possibly as a result of treated quiescent classic lesions that relapsed over time.

A vaccine for hepatitis C would likely not eliminate the risk of transmission, but a new study suggests it would dramatically reduce transmission rates among drug users from 90% to 25% or less.

Researchers from the University Hospital Zurich in Switzerland find that food seems to be a relevant trigger factor for abdominal angioedema attacks in patients with HAE.

Researchers from the Washington University School of Medicine have discovered an investigational therapy that could potentially benefit patients with an inherited form of ALS.

The development of new tools to quantify the many ways that rare diseases like FCS can affect patients is imperative.

Overweight and obesity in people with HIV are linked to behavioral factors and longer lives, not adverse effects of antiretroviral therapy, according to a new study.

In prior studies, younger populations that have asthma were not found to be impacted by autonomy preferences.

Investigators explore if polio can be used as a potential treatment for glioblastoma, an aggressive form of cancer that currently has no cure.

PAL-003 phase 2 extension study of long-term pegvaliase treatment in patients with phenylketonuria demonstrates substantial efficacy in maintaining reduced blood Phe concentration.