Rare Diseases
Conference Coverage
Burosumab is a ‘game changer,’ effective in all subgroups of XLH
The recently approved treatment improved serum phosphorus and key measures of pain and stiffness in subgroups of adults with this rare form of...
Feature
CDER chief reflects on advances in rare diseases
“People always call me fearless, but I feel like I just state the facts,” Dr. Janet Woodcock said.
News
Terlipressin squeaks by FDA review for hepatorenal syndrome 1
The FDA advisory committee’s 8-7 vote favoring approval of terlipressin for hepatorenal syndrome type 1 reflected concerns and misgivings.
News
FDA approves first oral somatostatin analog for acromegaly
The oral drug octreotide offers an alternative to long-term injections for the rare chronic disease.
News
First reported U.S. case of COVID-19 linked to Guillain-Barré syndrome
The unique clinical features in the U.S. case are urinary retention secondary to dysautonomia and ocular symptoms of...
News
Two rare neurologic conditions linked to COVID-19
Two variants of Guillain-Barré syndrome—Miller Fisher syndrome and polyneuritis cranialis—have been linked with COVID-19 infection.
News
First case of COVID-19 presenting as Guillain-Barré reported
A case report from China suggests a possible association between GBS and SARS-CoV-2 infection.
News
Osilodrostat gets FDA go-ahead for Cushing’s disease in adults
The oral cortisol synthesis inhibitor is the first therapy approved by the agency to address the overproduction of cortisol.
From the Journals
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
RNA inhibitors silence two new targets in dyslipidemia
PHILADELPHIA –
From the Journals
Juvenile dermatomyositis derails growth and pubertal development
Delayed puberty and growth failure were observed in children with juvenile dermatomyositis.