News

A peer-reviewed manuscript outlines the preclinical validation for the use of QR-110 in the treatment of Leber’s congenital amaurosis 10 (LCA10).

Obsessive compulsive disorder is commonly treated with medicines, psychotherapy, or some combination of the pair. It currently affects about 1% of US adults.

The FDA approved the 2 VMAT-2 drugs for the treatment of tardive dyskinesia in 2017.

A modified 12-week dosing schedule is now available for physicians administering the vascular endothelial growth factor (VEGF) inhibitor to patients with wet age-related macular degeneration.

The SPRINT endura and extensa are cleared for both chronic and acute pain including post-operative pain and post-traumatic pain.

The FDA granted an orphan drug designation to Rafael Pharmaceuticals, Inc.’s CPI-613 for the treatment of peripheral T-Cell lymphoma (PTCL).

The FDA has approved lenvatinib capsules (Lenvima, Eisai Inc.) for first-line treatment of patients with unresectable hepatocellular carcinoma.

For children whose mothers had high levels of a DDT metabolite, the odds of developing autism were one-third higher, and the odds of autism with intellectual disability were double.

A generic version of the 0.3 mg- and 0.15 mg-strength injectors has been approved for the treatment of patients that include those with life-threatening allergic reactions.

New research finds patients hospitalized for alcohol dependence have lower rates of survival if they are also infected with hepatitis C.

Experts argue that oral corticosteroids should be available at home for early use in asthma exacerbation.

The FDA has granted an orphan drug designation and a rare pediatric disease designation to Wave Life Sciences Ltd.’s WVE-210201 for the treatment of Duchenne muscular dystrophy (DMD).

The celebrity chef-turned-producer wants to champion healthcare access, both on the screen and in person. Where does he start?

The results of a new study point to age-related macular degeneration, diabetic retinopathy, and glaucoma as potential early warning signs that an individual is at risk for developing Alzheimer disease or dementia.

The approval is based on data from an ongoing phase 3 open-label safety study of 25 children with CF who have one of 10 mutations in the CFTR gene, in which ivacaftor demonstrated a consistent safety profile.

The FDA has approved ivacaftor (KALYDECO) as the first and only medicine to treat the underlying cause of cystic fibrosis in children ages 12 to

This is the first time that more than one gene has been implicated as the cause of spondylocarpotarsal synostosis syndrome.

The guidelines from the American Academy of Neurology highlight the need to improve diagnosis and ongoing rehabilitative care for patients with disorders of consciousness.

In an effort to reprogram retina cell regeneration, investigators activated dormant stem cells then aided other stem cells in developing into rod photoreceptor cells—the most abundant cell type in the retina which first aid the retina in sensing light.

Relatives of individuals with certain types of blood cancer may harbor inherited genetic changes that could influence their risk of developing diseases that arise from myeloid cells.

The phase 2 STARS trial evaluating OV101 as a treatment for Angelman syndrome achieves its primary endpoint of safety and tolerability.

The findings support exploration of LAC as a biomarker and therapeutic target that might help guide individualized treatments in the spirit of precision medicine.

A post-hoc analysis of data from the TREX-AMD trial has determined that eyes with neovascular age-related macular degeneration and treated with ranibizumab, develop more macular atrophy lesions within baseline choroidal neovascularization regions than outside those regions.

A review of the research on eHealth and mHealth interventions for youth with HIV concludes these types of interventions should be scaled up and more widely implemented.

The 30-person panel, including physicians and patients, recommended a median 12.5 tablets of 5mg opioid for 20 common procedures.

This combination of telmisartan, amlodipine, and chlorthalidone reduced blood pressure to target levels in 69.5% of patients at 6 months, compared to 55.3% of patients receiving usual care.

The first patients have been treated in a phase 2 trial evaluating ALLN-177 in adults and adolescents with primary hyperoxaluria or enteric hyperoxaluria with advanced chronic kidney disease (CKD) and elevated plasma oxalate.