Rare Diseases
Conference Coverage
Cilofexor passes phase 2 for primary biliary cholangitis
BOSTON – The nonsteroidal farnesoid X receptor agonist can reduce serum levels of liver biomarkers in patients with...
Conference Coverage
Families face challenges of gene therapy
Patients and manufacturers continue on a learning curve to improve trial designs and patient experiences.
Conference Coverage
Experts address barriers to genetic screening
WASHINGTON – Panel explores clinical implications of removing barriers to genetic testing for newborns and carriers.
Conference Coverage
Rituximab bests mycophenolate in pemphigus vulgaris
MADRID – “Rituximab has a superior overall benefit/risk profile, compared to mycophenolate mofetil, in patients with moderate to severe pemphigus...
Conference Coverage
C-Path and NORD team up to speed development of treatments for rare disorders
ROCKVILLE - A new data analytics platform aims to accelerate therapies from bench to bedside for rare diseases.
From the Journals
USPSTF: Screening pregnant women for asymptomatic bacteriuria cuts pyelonephritis risk
But the new recommendations urge clinicians to refrain from testing asymptomatic nonpregnant women and adult men.
From the Journals
New genotype of S. pyrogenes found in rise of scarlet fever in U.K.
From the Journals
Can we eradicate malaria by 2050?
A Lancet Commission report details a series of modeled projections that could point the way.
From the Journals
Possible role of enterovirus infection in acute flaccid myelitis cases detected
High levels of enterovirus peptides were found in the cerebral spinal fluid of individuals with AFM.
Latest News
Favorable Ebola results lead to drug trial termination, new focus
News from the FDA/CDC
FDA advisors recommend nintedanib for SSc interstitial lung disease
The positive outcome in nintedanib’s pivotal trial and prior FDA approval for idiopathic pulmonary fibrosis swayed panel members.