Rare Disease Advisor

FDA Clears Phase 1b/2 Trial of CD20/BCMA CAR T-Cell Therapy in Progressive Multiple Sclerosis

AbelZeta Pharma announced that the US Food and Drug Administration (FDA) has accepted its Investigational New Drug ...
Rare Disease Advisor

Cenobamate Shows Broad Seizure Control in LGS, Other DEEs, but Limited Benefit in DS

Cenobamate is effective in real-world use in LGS and other DEEs, but its risk-benefit profile varies with seizure type and ...
Rare Disease Advisor

IPF Progresses Faster Than Asbestosis, Study Shows

IPF progresses faster than asbestosis based on quantitative high-resolution computed tomography image analysis.
Rare Disease Advisor

P4O2-ILD Trial Protocol Targets Early PPF Biomarker Discovery

A new study will compare the different subtypes of ILD in terms of the rate and timing of progressive pulmonary fibrosis (PPF ...
Rare Disease Advisor

Elritercept Delivers Durable Transfusion Independence in Lower-Risk MDS

Elritercept produced rapid, durable transfusion independence and erythroid responses in patients with lower-risk MDS.
Rare Disease Advisor

Fenfluramine Regimens for Dravet Syndrome Linked to Lower Seizure Burden Than Other ASMs

Patients with DS at an Italian hospital whose treatment regimens included fenfluramine had better outcomes than those on ...
Rare Disease Advisor

Cerebrospinal Fluid TNFRSF8 Tracks With Huntington Disease Progression

In a study of Huntington disease (HD) carriers, CSF levels of TNFRSF8 tracked with both clinical progression and CAG repeat ...
Rare Disease Advisor

Rentosertib Treatment Shown to Reverse Biological Age in Patients With IPF

In a recent clinical trial, patients with IPF treated with the investigational therapy rentosertib exhibited a lower ...
Rare Disease Advisor

Can Warehouse Stores Bring Down the Cost of Health Insurance?

As the cost of living keeps rising, I’ve been turning to big box stores to try to save a few bucks. It’s not only food and ...