News

Xeris announced this morning that the U.S. FDA has granted Orphan Drug designation to the ready-to-use, liquid-stable glucagon for the treatment of Hyperinsulinemic Hypoglycemia

Teva has announced the release of a generic version of Syprine (trientine hydrochloride) capsules, 250 mg, as a substitute treatment for patients with Wilson’s disease in the United States.

A video game-like interactive program evidences promise in proof-of-concept study prior to FDA review as a medical device to treat children with ADHD.

The International Foundation for Functional Gastrointestinal Disorders will participate in Rare Disease Day on February 28, with efforts to support patients affected by a rare digestive disease globally.

New research shows patients whose immune cells have high levels of a particular protein face a higher risk of infection and a faster pace of disease progression.

Abeona Therapeutics, Inc. announced that the FDA has granted Orphan Drug Designation to its ABO-202 program for the treatment of infantile Batten disease.

New data from a 6-month, multicenter, observational study supplied evidence that DEX implants are safe and effective in real-life practice.

Amicus announced that the U.S. FDA has accepted its New Drug Application submitting for filing under priority review for migalastat for the treatment of patients with Fabry disease with amenable mutations.

How a self-sustaining system of medical meetings draws experts and future leaders together.

New research has shown that increased IOP is tolerable to corneal endothelium, and safe for most patients’ eyes despite immediate increases post-injection.

Reducing delays can help stop the spread of the infection and initiate treatment to patients quickly.

Data suggest that some gay and other men who have sex with men are using antiretroviral medications without a prescription.

The orthopedic surgeron from Northwell Health discussed the expectations of physically active older patients undergoing care for joints and knees.

A study revealed that utilizing ride-sharing apps like Uber and Lyft only resulted in a 0.2% lower rate of missed primary care appointments.

Enhanced protocol patients had $1,717 less total direct hospital costs versus the other patient group, translating to savings of greater than $250,000 annually.

DAAs have made the risk of HCV infection both manageable and ethically acceptable.

When a parent had T1D, a child is nearly 30% more likely to receive an ADHD diagnosis, though scientists have yet to figure out exactly why.

A new review of the scientific literature suggests there’s ample evidence over-the-counter painkillers have psychological effects.

Novo Nordisk has announced that their hemophilia B treatment, Rebinyn, is now available in the United States.

A collaborative research effort between the National Institute on Aging (NIA), the National Institutes of Health (NIH), and ChromaDex has found Niagen effective in preventing neurological damage and improving cognitive and physical function in a mouse model of Alzheimer’s disease.

The overall risk of asthma in childhood increases with maternal use of proton pump inhibitors and histamine-2 receptor antagonists.

The Amplify Hope Study, sponsored by the Rare Genomics Institute, analyzed the effects of educating families affected by rare disease on how to effectively crowdfund to pay for necessary genetic testing.

The label update for secukinumab (Cosentyx, Novartis) now includes the treatment for moderate-to-severe scalp psoriasis.

For the first time, it was shown that ibuprofen could be transferred to the baby through the placenta during this early phase of pregnancy

The researchers only needed to correct from 30% to 50% of the cardiomyocytes to rescue the mutant phenotypes and restore near-normal function.

Despite effective cART, HIV can hide in the spleen of patients with no detectable HIV in the blood.

The once-daily tablet regimen was approved for the treatment of HIV-1 infection. It is the smallest INSTI-based triple-therapy STR available.

The indication for letermovir represents a significant milestone, since CMV infection remains the most common complication of stem cell transplantation for patients with cancer.

Direct-acting antiviral agent treatment of HCV was well tolerated and appeared synergistic with chemotherapy of comorbid non-Hodgkin's lymphoma.

A new study finds patients can achieve long-term reductions in depressive symptoms through tele-psychotherapy.