Rare Diseases
Conference Coverage
CDC: Look for early symptoms of acute flaccid myelitis, report suspected cases
The average age of a patient diagnosed with AFM is 5 years
Conference Coverage
Mutant huntingtin and neurofilament light are potential biomarkers in Huntington’s disease
PHILADELPHIA – The biomarkers successfully distinguish between healthy controls, patients with premanifest disease,...
Conference Coverage
Pazopanib extended PFS in patients with carcinoid tumors
CHICAGO – Significant efficacy results need to be viewed in light of increased toxicity.
FDA/CDC
FDA approves Zolgensma for infantile-onset SMA treatment
Zolgensma is the first gene therapy for the treatment of infantile-onset spinal muscular atrophy in children aged less than 2 years.
Conference Coverage
2018: A banner year for hematology drug approvals
SAN DIEGO – There have been 32 new hematology drug approvals and the year isn’t over yet.