News

Thanks to an outpouring of support from the congenital muscular dystrophy (CMD) community over the past year, Cure CMD was able to announce new grant funding for 2018-2019.

The guidelines provide best-practices for physicians in these situations, though many physicians may be hesitant to provide aid during in-flight emergencies.

Data recently published in the Orphanet Journal of Rare Diseases showed that long-term improvements and stability can be achieved with taliglucerase alfa in patients with Gaucher disease.

The medication has also been linked to increased risk of autism and reduced IQ.

George Yohrling, Ph.D., Senior Director of Mission and Science at the HDSA discusses the challenges that come with working with patients with HD, and communicating with their family members and caregivers.

15-year old biliary atresia patient Jarrius Robertson will be honored by the WWE. At the 2018 WWE Hall of Fame Induction Ceremony on April 6, he will become the fourth recipient of the Warrior Award.

Children who had long-term exposure to 'greener' neighborhoods had higher brain volume in several regions. Additionally, peak volumes in some of the clusters associated with greenness also predicted better scores for cognitive tests.

On Rare Disease Day 2018, Shire plc will focus on promoting prompt diagnosis of rare diseases, as it is the most important issue affecting health, longevity and well-being for patients and their families..

Positive outcomes were reported from a biomarker study evaluating intracellular glycosaminoglycans (GAGs) levels in leukocytes as a disease activity biomarker in MPS VI.

This meta-analysis does not provide support for current recommendations to use omega-3 fatty acid supplements for the prevention of cardiovascular disease risk.

In combination with low-dose hormone therapy, elagolix reduced heavy menstrual bleeding in 68.5% of women with uterine fibroids.

They were also 3 times more likely to suffer from cancer, and reported a greater rate of hypertension and cardiovascular disease.

The National Center for Advancing Translational Sciences and the National Institutes of Health Clinical Center will honor World Rare Disease Day this year by hosting an event to celebrate patient advocacy.

Last week in Nature, data were published revealing that AD-114, a locally developed molecule, could be a therapeutic option for patients with idiopathic pulmonary fibrosis.

Fortress Biotech and its subsidiary Aevitas Therapeutics have entered into a sponsored research agreement with Dr. Guanping Gao’s laboratory at the University of Massachusetts Medical School.

This morning, Children’s National Health System and Horizon Pharma plc have announced the creation of the Horizon Pharma Clinical Care Endowment at the Children’s National Rare Disease Institute.

Results demonstrate a statistically significant improvement in ongoing pregnancy benefit at 10 weeks following a single, oral dose of nolasiban.

On Rare Disease Day, a tribute committee will rename a plaza in Cambridge, Massachusetts in honor honor of Henri A. Termeer, late biotechnology pioneer and former Genzyme Chief Executive Officer.

Medtronic had both its Guardian Sensor 3 CGM system and it's Resolute Onyx 2.0 mm drug-eluting stent approved by the FDA.

John Crowley, the President and CEO of Amicus Therapeutics, sat down with Rare Disease Report at the 14th Annual WORLDSymposium in San Diego to discuss the President's mention of rare diseases.

The Committee for Medicinal Products for Human Use of the EMA recommended that both Mylotarg (gemtuzumab ozogamicin) and Bosulif (bosutinib) be reviewed separately by the European Commission.

Those in deprived populations—the homeless, those with substance abuse disorder, sex workers, and prisoners—have a significantly higher rate of all-cause mortality than their counterparts.

For medical school students, choosing a specialty is a decision that carries a lifetime's worth of weight. What factors go into that choice?

Recruiting risk networks more efficiently finds higher proportions of undiagnosed HIV-positive people.

Results indicate novel activation of CD4+ T-cells among HIV-infected youth who have STIs aside from HSV, contributing to disease progression.

Most of the high-risk HPV types detected were included in the 9v vaccine, impacting clearance rates by decreasing the overall burden of infection in HIV-infected women.

A new method of manufacturing HIV vaccines holds the promise of speeding up the process and lowering the cost.

The monoclonal antibody therapy was used on a limited basis during the 2014 West Africa Ebola outbreak, despite not being marketed at the time.

Apadaz is the first prodrug of hydrocodone and acetaminophen to be FDA approved.

Sobi announced that the EMA's Committee for Medicinal Products for Human Use has adopted a positive opinion for anakinra (Kineret) for the treatment of Still’s disease.