News

Plazomicin (Zemdri, Achaogen) is designed to treat certain multi-drug resistant complicated urinary tract infections in adults.

The FRESCA Medical, Inc system is designed to operate with less airflow, alleviating some of the complaints that arise with traditional CPAP systems for patients with obstructive sleep apnea.

In a study conducted at Children's Hospital Los Angeles (CHLA), less-toxic treatment regimens for mixed phenotype acute leukemia (MPAL) have been found to coincide with disease remission.

Cannabidiol (Epidiolex) is the first drug containing a marijuana-derived substance to receive FDA approval.

The device offers a new treatment option for surgeons managing glaucoma patients’ intraocular pressure.

The FDA has approved cannabidiol (Epidiolex) [CBD] oral solution in patients aged 2 years and older for the treatment of severe and rare types of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome.

The FDA has granted Priority Review for a supplemental New Drug Application (sNDA) for the company’s Waldenström's macroglobulinemia combination therapy, ibrutinib (IMBRUVICA) in combination with rituximab (RITUXAN).

Children born early preterm (gestational age 22-33 weeks) scored an average of 0.24 SD higher than children born full term. For girls, this increased to 0.8 SD higher than term born sisters.

Firstborn children with ≥2 younger siblings have a 1.71 times higher risk of developing ADHD compared to children without siblings.

Eligibility criteria for HIV pre-exposure prophylaxis (PrEP) based on behavioral markers does not capture all with biological markers for HIV risk.

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

The first patient has been enrolled in a pivotal phase 3 clinical trial evaluating the safety and efficacy of an epithelium-on (epi-on) corneal collagen cross-linking procedure to treat progressive keratoconus.

The authorization of these 2 devices allows for less-invasive vascular access options for patients requiring hemodialysis.

This is the only long-acting local anesthetic to demonstrate significantly reduced postoperative pain and opioid use through 72 hours compared to bupivacaine solution for postoperative pain management.

The rare condition affects the skin and can lead to ulceration and scarring, however, there are currently no FDA approved treatments for necrobiosis lipoidica.

The FDA has granted Orphan Drug Designation to its product, PCS499, for treatment of necrobiosis lipoidica.

In the phase 1b trial, one-time administrations of VY-AADC demonstrated robust and durable improvements in patients’ motor function.

Positive preliminary results from the Sarepta Therapeutics, Inc phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin vector in individuals with Duchenne muscular dystrophy (DMD) indicate outstanding promise.

Impel NeuroPharma will study the safety and tolerability of dihydroergotamine delivered through the company’s Precision Olfactory Delivery device.

Evidence determines that those receiving treatment for nAMD should delay cataract surgery within 6 months of starting treatment, if possible.

HIV-positive patients who take antiretroviral therapy have a lower risk of cancer than HIV-positive patients who are not taking antiretroviral drugs.

The system’s algorithm predicts glucose levels 30 minutes in advance and adjusts insulin delivery to reduce hypoglycemic events.

NOCDURNA is the first and only sublingual tablet indicated for treatment of nocturia due to nocturnal polyuria in adults awakening at least 2 times per night to urinate.

hATTR compass program will empower people with accurate genetic information so they can make informed decisions about their healthcare.

The Eversense CGM System sensor is implanted into the patient’s arm and sends glucose data every 5 minutes to a mobile app.

Researchers identified 6 types of potentially inappropriate prescriptions, all of which were associated with higher hazards for all-cause mortality, 4 with non-fatal overdose, and 5 with fatal overdose.

The FDA has approved a label expansion for C1 esterase inhibitor [human] (CINRYZE) as a preventative measure for pediatric angioedema attacks in children aged 6 years and older with hereditary angioedema (HAE).

Nicholas J Robert, MD, discusses treatment challenges associated with rare cancers like Merkel cell carcinoma.

This is the first and only therapy indicated in the US to help prevent angioedema attacks in pediatric patients as young as 6 years old.

Researchers noted that a reduction in the frequency of medication regimen has been proven to also aid treatment adherence.