Rare Diseases
News
Zilucoplan improved efficacy outcomes in myasthenia gravis
The complement C5 inhibitor had a similar magnitude of clinical effect as that seen in eculizumab studies.
FDA/CDC
FDA approves first treatment for advanced epithelioid sarcoma
Tazemetostat earns accelerated approval for the treatment of patients aged 16 years and older with metastatic or locally advanced epithelioid...
News
Expedited review programs not shortening drug development
While it may be hard to understand the impact of the many expedited review programs introduced at the FDA, there is still room for improvement in...
Conference Coverage
High complete response rate seen with novel CAR-T for myeloma
ORLANDO – A CAR T product targeting B-cell maturation antigen, one of several in development, was associated with a 100% overall response rate.
Conference Coverage
Experts address barriers to genetic screening
WASHINGTON – Panel explores clinical implications of removing barriers to genetic testing for newborns and carriers.
News
Neurologists publish consensus statement on stridor in MSA
The statement provides guidance on the diagnosis, prognosis, and treatment of stridor in patients with MSA, as well as indicating areas for future...
FDA/CDC
FDA approves afamelanotide for treatment of rare condition with light-induced pain
This is the first treatment approved to help patients with erythropoietic protoporphyria increase their exposure to light.
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.
News
New genotype of S. pyrogenes found in rise of scarlet fever in U.K.
Conference Coverage
‘Pot’ is still hot for Dravet, Lennox-Gastaut
BANGKOK – Long-term extension studies show sustained, consistent seizure reductions.