News

Kazia Therapeutics has received orphan drug designation by the U.S. FDA for GDC-0084, intended for the treatment of glioblastoma multiforme.

Study data revealed that the patients who received a 2-week course of the antibiotic observed an unexpected increase in death.

At the Cost-Sharing Roundtable on Friday, K. Robin Yabroff, Ph.D. presented data explaining the economic struggles that can be had by those who need medical attention.

A new paper argues that the persistent immune activation associated with HIV leads to a heightened risk of cardiovascular disease.

Preliminary study results showed that Fecal donor-unrelated donor mix for initial Clostridium difficile infection is associated with specific bacterial communities that do not resemble the donors’ sample.

A new study looked at the social links that underpin HIV infection risk among people who inject drugs.

After WORLDSymposium, Rare Disease Report spoke with Loren D.M. Pena, M.D., Ph.D., who presented new safety data for neoGAA in late-onset Pompe.

A walking test accurately diagnosed 97% of the time between 2 causes of dementia.

Vertex has initiated a Phase 3 study of VX-659, Tezacaftor and Ivacaftor for people with cystic fibrosis who have one F508del mutation and one minimal function mutation.

In the early stages of the FCX-007 program, no children were included. Fibrocell has been testing the potential therapy in adults until enough successful data were collected to justify treating the pediatric population.

The study authors determined that while the videos can be beneficial, health professionals should be attuned to the content to ensure what is consumed is not inaccurate or misleading.

ContraVir Pharmaceuticals, Inc. announced that the U.S. FDA has granted orphan drug designation to TXL, an investigational drug candidate for the potential treatment of chronic hepatitis B infection in a pediatric patient population.

Black patients had a 62% greater chance of having experienced an ischemic stroke pre-AF diagnosis, and a 67% higher risk of having one post-AF diagnosis.

The new, short screening scale detects the vast majority of general population cases at threshold and can be used as a screening tool in specialty treatment settings.

“Original antigenic sin" is the result of slight year-to-year virus change that are not caught by antibodies produced by the body during previous vaccinations.

The CDC's committee voted unanimously to support the vaccine, widening its availability to patients through health care coverage and plans.

The FDA has accepted a supplemental Biologics License Application (sBLA) for Shire’s Cinryze, a therapy that treats recurring swelling attacks in hereditary angioedema (HAE), to expand the already approved indication to include children 6 years and older.

Soleno Therapeutics has completed its End-of-Phase II meeting with the FDA regarding the biopharmaceutical company’s drug, Diazoxide Choline Controlled-Release (DCCR), a therapy used to treat patients with Prader-Willi syndrome (PWS).

Findings suggest cannabinoids may have an immunological benefit, limiting the risk of development of non-AIDS-associated comorbidities.

GenSight Biologics has published successful results in the Journal of the American Academy of Ophthalmology from their Phase I/II clinical trial and long-term follow-up of GS010, a therapy used to treat patients with Leber Hereditary Optic Neuropathy (LHON).

Weight from age 25 onwards has been linked to the risk of more or less heart damage, as measured by levels of the protein troponin, underscoring the importance of long-term weight control.

New data from the US Centers for Disease Control and Prevention show that robust asthma education and prevention programs are working.

The collaboration intends to combine AbbVie’s clinical resources in monoclonal antibody develop with Voyager’s gene therapy platform.

The NeoTract product was originally indicated to treat those with BPH aged 50 years and older.

In addition to the outbreak, kratom is expected to have a similar risk of addiction, abuse and death as an opioid, the agency announced earlier this month.

In a story written by Jackie MacMullan and published on ESPN.com, it was revealed the Hall of Fame basketball player Nate “Tiny” Archibald has been living with amyloidosis.

This is the first tentative approval of tenofovir alafenamide, coming just 2 years after the FDA approved Gilead Sciences' combination treatment of emtricitabine and tenofovir alafenamide.

Shire plc, Microsoft and EURORDIS announced a strategic alliance with the intentions of accelerating the time it takes for pediatric rare disease patients to receive the correct diagnosis.

The study finds that general psychopathology is a heritable trait in youth which could be detected early in life through brain structural connectivity.

The American Lung Association will host two events this spring: the 2018 Greater Philadelphia LUNG FORCE Expo and a webinar for sarcoidosis patients focusing on improving health outcomes and support programs.