News

A review of more than 75 studies has revealed a link between persistent T cell activation and multiple risk factors for CVD.

The gammaCore device was previously approved for the acute treatment of pain associated with episodic cluster headaches in adult patients.

PTC Therapeutics has announced that Part 1 of the FIREFISH study has shown that RG7916, a Type 1 spinal muscular atrophy (SMA) drug, to be safe and well-tolerated in infants and babies with Type 1 SMA.

Emmaus Life Sciences has announced that it has a Medicaid Drug Rebate Agreement with the Centers for Medicare and Medicaid Services, allowing coverage of Endari (L-glutamine oral powder).

The therapy is approved for the treatment of HIV-1 infection in adults and pediatric patients ages 12 years and older.

The Canadian Agency for Drugs and Technologies in Health organization’s Common Drug Review protocol often leaves rare disease patients in British Columbia without governmental payment coverage for their expensive treatments.

As the Myanmar military’s genocidal actions continue to displace Rohingya refugees, the persecuted minority group is experiencing the largest diphtheria outbreak since the 1920s.

This morning, Abeona Therapeutics announced that the U.S. Food and Drug Administration granted EB-101, its gene therapy for epidermolysis bullosa, Regenerative Medicine Advanced Therapy designation.

The oral liquid therapy is expected to improve patient access and reduce pharmacist burdens by no longer compounding liquid formulations.

This morning, Aradigm announced that it received a Complete Response Letter, denying its New Drug Application for Linhaliq.

The percentage of women who filled at least 1 prescription rose 344% from 2003 to 2015.

Many physicians find their motivation waning as the pressures of life continue to build. How can doctors reach retirement on their own terms?

A review of antidepressants' impact on PLWH with depression suggested that they should be utilized for patients with HIV—a population at high risk for depression.

The protein functions by regulating bacteria’s gene expression, thereby affecting its flagella motion, protein consumption, and production of biofilm to attach to hosts.

Alnylam announced that its Marketing Authorisation Application for patisiran was accepted by the European Medicines Agency.

Novartis has received U.S. FDA approval for lutetium (177Lu) oxodotreotide (Lutathera) for the treatment of somatostatin receptor positive GEP-NETs.

New data concludes that neurofeedback training could potentially increase brain connectivity in patients with Huntington’s disease (HD).

The international study will compare the World Health Organization's recommended regimen against 2 others.

Cases of sSAH in pregnant women have increased 2%, while less than half of all stroke patients are receiving proper rehabilitation services post-discharge.

The latest #ALSPepperChallenge video comes from Rare Disease Report. Participate today to raise awareness for ALS.

Rare Disease Report sat down with acute intermittent porphyria patient Nichol Kirby who was diagnosed in 2015 and accused of being a drug seeker looking to feed an opioid addiction.

All regions included in the analysis experienced notable increases in pretreatment NNRTI resistance each year.

New research investigating a novel means to eradicate HIV found a significant roadblock on the way to a cure.

The peer-reviewed clinical journal Neurology has published the Phase 2 trial results for arimoclomol in patients with SOD1 amyotrophic lateral sclerosis (ALS).

While maintaining contact precautions for extended periods can keep from spreading, unnecessarily long isolation can negatively impact care.

For primary care to increase transformation efforts integrating survivorship services into routine care, survivorship must become a recognized clinical category.