Study Finds Repeat Dosing of IVIG and PLEX Common in GBS, Resulting in Harm to Nonresponders

According to randomized controlled trials, repeat intravenous immune globulin (IVIG) dosing and plasma exchange (PLEX) followed by IVIG (combination therapy) have no additional therapeutic benefit in Guillain-Barré syndrome (GBS) nonresponders. Furthermore, the delineation between GBS and acute onset chronic inflammatory demyelinating polyneuropathy (A-CIDP) can be particularly challenging and carries therapeutic implications.

Investigational Plasma Kallikrein Inhibitor (Sebetralstat) Reduces Time to Relief from Hereditary Angioedema Attacks

In a Phase III, double-blind, three-way crossover trial, sebetralstat given on-demand for treatment of hereditary angioedema (HAE) attacks was shown to result in faster times to the start of symptom relief, reduction in attack severity and complete attack resolution than placebo treatment.

How FDA Is Working to Accelerate Rare Disease Treatments

Through regulatory pathways and patient engagement, FDA is helping to advance treatment innovations for rare diseases.

Solving Clinical Trial Delays by Accelerating Patient Recruitment

Enrolling patients in clinical trials is a slow and costly process, but software companies are working to address the problem.

First Cancer Patients Receive mRNA Therapy in Clinical Trial

Imperial College Healthcare NHS Trust has enrolled the first United Kingdom (UK) patients who have received an experimental messenger RNA (mRNA) therapy — a type of immunotherapy treatment called mRNA-4359 — in its Phase I/II clinical trial.

First Do No Harm: Increasing Patient Safety

Patient safety is at the heart of healthcare, so it’s imperative to put in place systems and regulations to reduce patient harm.

Efgartigimod Efficacious in Treating Chronic Immune Thombocytopenia

A new study shows efgartigimod significantly increased sustained platelet count responses compared with placebo in patients with chronic immune thrombocytopenia, including those who had received multiple previous immune thrombocytopenia therapies.

Study Finds Experimental Alzheimer’s Drug Slows Cognitive Decline

A large clinical trial of Eli Lilly’s experimental Alzheimer’s medication, donanemab found the drug slowed declines in patients’ ability to think clearly and perform daily tasks by more than a third.

NIH Provides $5.3 Million Grant to Study Down Syndrome

The first clinical trial for Down syndrome regression disorder is being funded by a five-year $5.3 million grant.

First Human Clinical Trial of Cancer Killing Virus Has Begun

A clinical trial of the Vaxina (the CF33-hNIS virus) designed to kill cancer cells has begun.