News

New research finds that the prevalence of opioid use remained high during the study period from 2007 to 2016.

The FDA has granted an orphan drug designation to ArmaGen Inc.’s GT-184 for the treatment of mucopolysaccharidosis type IIIA.

Investigators from Milan, Italy, have identified an important molecule involved in the onset of systemic scleroderma.

Times are tough. Medicine needs a hero.

The FDA has granted a breakthrough therapy designation to Daiichi Sankyo Company, Limited’s investigational FLT3 inhibitor, quizartinib, for the treatment of adult patients with relapsed/refractory FLT3-ITD acute myeloid leukemia (AML).

Research suggests that elevated glycemia, low blood pressure, and low BMI may be identifying markers for dementia risk and possible targets for management or prevention.

The combined results of a recent meta-analysis and a new population-based study supports there being a significant association between asthma and ADHD.

Phase 3 trial assessing RHB-104 as a treatment for Crohn’s disease yields positive results.

High levels of the protein Mfn2 were found to prevent nerve degeneration, muscle atrophy, and paralysis of amyloid lateral sclerosis (ALS) in a mouse model.

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 long-acting injectable risperidone (Perseris) will provide an alternative to daily oral medication to improve treatment adherence for patients with schizophrenia.

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.