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The fixed-dose combination therapy from Valeant Pharmaceuticals International is vying to become the first and only approved topical lotion for the condition.

The next generation platform measures the eye inside and out to enable highly precise custom laser vision correction.

Although there were no concerns regarding the safety profile of the CGRP inhibitor, the reduction in cluster headache attacks from baseline over the 12-week treatment period was underwhelming.

The first participant was dosed in BioMarin Pharmaceutical Inc.’s global phase 2 study for vosoritide for the treatment of infants and young children with achondroplasia.

New evidence suggests that Rasmussen’s encephalitis is an autoimmune disease.

A new Australian study found overall condom use decreased when PrEP was introduced into the region.

Those discharged early and administered rivaroxaban saved $2496 and spent 26.4 fewer hours hospitalized over the course of 90 days.

Obstructive sleep apnea could be the cause of fatigue in many people with HIV.

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

A new analysis shows fully one-third of patients prescribed direct-acting antivirals are denied coverage by their insurers. Denial rates are even higher for patients with private insurance.

How history shows us that changing dogma in healthcare practice requires a multi-faceted approach.

The FDA has granted Orphan Drug Designation to AMO-04 for the treatment of Rett syndrome.

Interim results from Novartis’ survey, I-WISh, have revealed high, negative impacts of the rare blood disorder, immune thrombocytopenia (ITP), on patients’ quality of life.

Steven Benner, MD, MHS, senior vice president and global therapeutic area head of Oncology, discusses the use of gilteritinib in patients with relapsed/refractory FLT3 mutation-positive acute myeloid leukemia (AML).

IgE for alpha-gal was identified in 26% of the patients, and plaque buildup was 30% higher in those patients compared to those that were not sensitized.

The 1300-plus patient survey interim results show patients struggle with fatigue and maintaining control of daily functions.

The buprenorphine and naloxone sublingual film is approved for the treatment of opioid abuse disorder in combination with counseling and psychosocial support.

Data revealed that the 1-year patency rate was 89.1% via Kaplan Meier estimate at day 360, and a CD-TLR rate of 7.1%.

The findings present the first assessment of peripheral changes in AMD and time to dark-adapt and could establish DA as a promising functional outcome measure in AMD.

Rhythm Pharmaceuticals, Inc. has completed enrollment for its phase 3 clinical trials for the evaluation of setmelanotide for pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesity.

The authors found no studies supporting the use of alternative treatments, such as cognitive behavioral therapy or omega fatty acids, over ADHD medications.

Though patients had a clinically meaningful increase in pre-bronchodilator FEV1, they did not evidence a significantly improved annualized asthma exacerbation rates compared with placebo.

It is the first approval of a product for this indication and is now available on Optovue’s Avanti Widefield optical coherence tomography system.

Researchers hope to move closer to finding a treatment for Duchenne muscular dystrophy, using new stem cell-based technology and genes edited with CRISPR.

The specifics of the "minor deficiencies" cited by the FDA will be relayed on June 27, 2018, upon the official issuance of the CRL.

A case of plague was reported in Elmore County, Idaho, in a young boy who is currently recovering with the administration of antibiotics.

In data assessed for the CE Mark, 70.6% of the patients experienced at least a ≥50% reduction in symptoms or a return to normal rates of bowel voidance at the 6-month mark.

ContraVir plans to begin a streamlined early clinical program to test the clinical safety and efficacy profile in healthy volunteers and HBV-infected patients.

A national discussion on a public health crisis is slowing down before it can even really start — again.

Children with amblyopia and strabismus took roughly 28% longer than their peers when transferring answers to a multiple-choice answer form, despite having good vision in 1 or both eyes.