News

Pivotal clinical trial, CONNECT-FX, commences to evaluate CBD gel as a treatment for fragile X syndrome.

An examination of trials supporting the 46 therapies approved via the Breakthrough Therapy designation has shown that fewer data supports their approval than what may be believed.

The antiviral treatment for patients at high risk of complications from influenza met the primary objective of efficacy measured by time to improvement of symptoms compared to placebo.

Researchers compared the cost-effectiveness of vancomycin, fidaxomicin, and bezlotoxumab with vancomycin as treatments for treating the first recurrence of Clostridium difficile infection.

Emixustat has been shown in previous preclinical analysis to slow the visual cycle and inhibit the activity of RPE65 and associated loss of photoreceptors associated with RPE dysfunction.

Positive data for the add-on therapy for patients with type 2 diabetes and chronic kidney disease will be presented at an upcoming medical meeting.

Study performed by researchers at Juntendo University reveals molecular mechanisms that trigger CALR-mutant myeloproliferative neoplasms (MPNs).

More treatments are needed for ALS, beyond the available riluzole and edaravone.

A recent study provides the first large-scale evidence that the cost of care for some rare pediatric skin conditions can potentially be reduced while achieving the same outcomes.

The Surpass Streamline stent is approved to treat unruptured large and giant wide-neck intracranial aneurysms.

Fecal microbiota transplant and oral metronidazole are equally effective and tolerated for C. difficile treatment.

Canadian study finds patient's age is a modest factor in HIV viral rebound after suppression with treatment.

Holidays and special events can cause increased likelihood of status epilepticus, possibly due to alcohol consumption and withdrawal, changes in sleep patterns, and non-compliance with anti-seizure drugs.

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

Where does current research focuses its resources, and what may the future of such research involve?

The medical director of the Baylor AT&T Memory Center explains how in-house biomarker testing could improve opportunities for clinical drug research.

The virus was deemed eradicated by the World Health Organization in 1980. But there remains public concern of its use as a bioweapon.

The FDA has approved the first drug indicated for the treatment of smallpox.

The phase 3 trial evaluating ZX008 as a treatment for Dravet syndrome met its primary endpoint.

The resubmitted biologics license application includes data from pivotal phase 3 studies of more than 11,000 patients.

A systematic review and meta-analysis reveals differences between VMAT-2 inhibitors for tardive dyskinesia in absence of head-to-head clinical trials.

AbbVie has submitted a supplemental new drug application to the FDA for venetoclax to be used in combination with HMA or LDAC for the treatment of AML patients who are ineligible for intensive chemotherapy.

The CDC finds that substantial numbers of persons unaware of their HIV infection report missed opportunities for earlier diagnosis.

Three-quarters of patients who discontinue antipsychotic treatment experience relapse within 12-18 months. However, there are no current clinical guidelines describing treatment continuation or discontinuation beyond 1-2 years.

A single session with a therapist significantly reduced depression for up to 3 months and dating violence perpetration for up to 12 months. The computer-based intervention reduced both for 6 months.

The FDA has granted orphan drug designation to Acceleron Pharma Inc for its product, ACE-083, for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

Results from a phase 3 clinical trial evaluating an ibrutnib combination with a chemotherapy regimen for the treatment of diffuse large B-cell lymphoma fails to meet primary endpoint.

Preliminary results from part 2 of the trial in patients with are expected in the second half of 2019.

The FDA has accepted and granted priority review for a new supplemental Biologics License Application (sBLA) for pembrolizumab (KEYTRUDA) for the treatment of advanced hepatocellular carcinoma (HCC).

The newly-revealed structure of the neurotransmitter GABAA, a γ-aminobutyric acid, opens new possibilities for drugs to treat epilepsy, anxiety, and insomnia.