News

Review corroborates sports medicine findings that athletes with ADHD are at risk for increased cognitive dysfunction and prolonged recovery from concussions.

New research suggests that there could be a possible connection between CTE and ALS.

Increased prevalence of HCV infection among hospitalized children is linked to increase in substance abuse

Shaquem Griffin, rare disease survivor and linebacker for the University of Central Florida football team, has received an invitation to the NFL combine.

A meta-analysis found methylphenidate treatment of children with ADHD improves academic productivity more than accuracy, effecting performance in math more than reading.

HCV reactivation can occur during treatment of non-hepatic cancer, increasing risk of hepatic injury and interfering with cancer treatment.

Alnylam has announced this afternoon that the U.S. FDA has accepted for filing its New Drug Application for patisiran, an investigational RNAi therapeutic targeting transthyretin for the treatment of hATTR amyloidosis.

AVROBIO, Inc. announced that it has completed a $60-million Series B financing, of which proceeds will be used to advance several gene therapies.

Amid a widespread flu season, one dentists offers reason to keep a check on vitamin D levels.

Vertex announced that VX-659 and VX-445 are being advanced into Phase 3 development as part of triple combination regimens for people with cystic fibrosis.

Rockefeller University scientists have observed the effects of Huntington’s disease in neurons as early as conception, and suggest therapies that block HTT protein activity may be doing more harm than good.

Treating adults with ADHD challenges clinicians to overcome associated stigma, complexity and controversy to help patients overcome this hidden disability.

Rare pediatric disease designation was granted by the FDA to MeiraGTx’s A002 (ZZV2/8-hCARp.hCNGB3) for achromatopsia.

The CDC attributes up to 21% of US asthma deaths to occupational exposures, and considers many potentially avoidable.

Physicians point to a murky history between buyers and PBMs to express doubt for the future of CVS’ acquisition of Aetna.

Zogenix announced that the last patient has been randomized into the treatment period of Study 1504, its second Phase 3 clinical trial evaluating ZX008 in Dravet syndrome.

About 1 in 7 postoperative lung resection patients are persistently using opioids after being introduced to them for recovery.

Results from a study recently published in the Orphanet Journal of Rare Diseases confirms the idea that tuberous sclerosis complex (TSC) can be diagnosed before seizure onset.

NBC News chief foreign correspondent Richard Engel's most demanding role has taken place off-camera. He and his wife, Mary Forrest, support each other in raising their 2-year old son, Henry, a patient with Rett syndrome.

Fitzgerald reportedly purchased stock in Merck & Co., Bayer, Humana, and the US Food Holding Co., after starting the role as CDC director.

The first patient was enrolled in Mallinckrodt’s Phase 4 pilot study to further assess the efficacy of H.P. Acthar Gel (Repository Corticotropin Injection) in patients with pulmonary sarcoidosis.

This morning, Aquestive Therapeutics, Inc. announced that the U.S. FDA has granted orphan drug designation to Riluzole Oral Soluble Film (riluzole OSF) for the treatment of ALS.

This is the first national study to analyze prescription rates of ADHD, anxiety disorders and depression psychiatric medications in youth.

A review of clinical trials has shown that GPi and STN targets can be successful in DBS, but all options should be considered based on the needs of the patient.

Multi Radiance is one of the few laser manufacturers in the world to secure the NHN product class FDA 510(k) clearance.

In an effort to raise awareness about the rare disease community, ORDI is organizing the Race for 7 run.

Amgen announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion for recommending a label variation for Kyprolis (carfilzomib).

This morning, Fibrocell Science, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted the company allowance to begin enrollment in the Phase 2 portion of its Phase 1/2 clinical trial of FCX-007.

Results from an observational case series published in the Orphanet Journal of Rare Diseases conclude that ezetimibe can be an effective, safe, and sustainable treatment option for LAL-D.

School-based sex education incorporating relevant information on condom use, PrEP and elevated HIV risk could reduce sexual risk behaviors and HIV transmission.