News

XYOSTED's approval comes nearly a year following Antares first received a Complete Response Letter from the FDA for its application.

The association between moderate hyperbilirubinemia and childhood asthma and lack of association with higher bilirubin levels indicates the association is not causal.

Zinc is the second most abundant trace element in the human body after iron, and is present in high concentration in the human eye.

It’s long been established that obesity can lead to asthma, but new research suggests asthma can also lead to obesity.

Upon finding pH and oxygen may influence how antibiotics perform in CF lung infections, investigators suggest a one-size-fits-all therapy approach may not be the best treatment.

Results from the interim analysis of the ENVISION phase 3 trial shows that givosiran treatment was associated with a statistically significant reduction in urinarly ALA levels in patients with acute intermittent porphyria.

Elbasvir/Grazoprevir has performed well in clinical trials, even among patients with HIV and other coinfections.

The FDA has approved amikacin liposome inhalation suspension (Arikayce) for the treatment of lung disease caused by Mycobacterium avium complex (MAC) in a limited population of patients with the disease who do not respond to conventional treatment (refractory disease).

There is a found significant unmet need to develop treatments that limit or protect against scar formation, GA development, retinal thinning, fluid increase, and lesions, as an accompaniment to anti-VEGF therapy.

Investigators stressed the importance of culturally competent care for patients with dementia and support for their caregivers.

The robustness of neural responses to positive and negative events may modify adolescent girls’ risk of depression, according to a new study.

The pulmonologist and senior medical advisor, American Lung Association, highlights the necessary steps of smoking cessation practices as well as the unknown surrounding e-cigarettes.

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

The new document will provide guidance on how and when the FDA intends to collect, compile, and publicize retail consignees that may have received recalled foods.

The new formulation, known as Xolair PFS, will be available in the US by the end of the year.

Perampanel (FYCOMPA) has been approved for patients aged 4 years or older, who suffer from partial-onset seizures with or without secondarily generalized seizures, as both a tablet and oral suspension formulation.

Updates in trivalent vaccines and vaccination clearance for egg-allergic patients were among the updated recommendations for pediatric influenza prevention and treatment this upcoming 2018-2019 flu season.

In a recent interview with Rare Disease Report®, Hartmann Wellhoefer, MD, discussed the biggest challenges faced and advances being made in the fight against MPS.

An interview-based qualitative study found that minority residents face extra burdens during training due to racial/ethnic bias and microaggressions.

Galcanezumab will be available as a monthly 120 mg injection in pen and syringe forms for the prevention of migraine in adults.

A 16,000-patient assessment found that infection alone, excluding critical illness, is associated with reduced brain volume later in life.

The FDA has approved a 0.15mg dosage of Symjepi, an epinephrine injection, for the emergency treatment of serious allergic reactions in children.

Robert J. Kreitman, MD, discusses the FDA’s recent approval of moxetumomab pasudotox (Lumoxiti) for the treatment of hairy cell leukemia (HCL).

Less than one-third received both influnza and Tdap vaccination during their pregnancies, citing limited understanding or assurance of their benefits and safety.

The FDA has granted an orphan drug designation to Q Therapeutics Inc.’s Q-Cell for the treatment of transverse myelitis.

The FDA recognized the potential of proCase, which is being developed to treat blood clots and does not increase the risk of bleeding.

How can caregivers improve their management of treatments—both in cost and in availability—for children in need of help due to asthma.

A modeling analysis suggests state officials in Indiana could have prevented upwards of 95% of the HIV cases in a 2014 outbreak.