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.
Plasma Prekallikrein Inhibition by Antisense Oligonucleotide Reduces Hereditary Angioedema Disease Burden

An investigational antisense oligo-nucleotide (donidalorsen) that acts by degrading plasma prekallikrein messenger RNA was evaluated in patients with hereditary angioedema (HAE) and C1 inhibitor deficiency to assess whether this agent can reduce the frequency of angioedema attacks and the burden of disease.
Many Clinically Stable CIDP Patients Can Safely Stop IVIG Maintenance Treatment

While intravenous immune globulin (IVIG) therapy is efficacious for patients with chronic inflammatory demyelinating polyneuropathy (CIDP), the lack of biomarkers for disease activity makes the need for ongoing treatment difficult to assess.
Addition of Eltrombopag Improves Efficacy of Standard Therapy for Aplastic Anemia

Eltrombopay found to improve therapy for aplastic anemia.
Neonatal Fc Receptor Blockers for Myasthenia Gravis: The Concept Is Now Reality

While acetylcholinesterase inhibitors, corticosteroids and thymectomy are generally considered first-line therapies for MG, many patients additionally require proven immunosuppressive therapies (ISTs) such as azathioprine, cyclosporine/tacrolimus or mycophenolate mofetil, or immunomodulatory therapies, including intravenous immune globulin (IVIG) or plasma exchange (PLEX).
IG Treatment May Reduce Acute Exacerbations of COPD

Observational studies suggest immune globulin (IG) treatment may reduce the frequency of acute exacerbations of chronic obstructive pulmonary disease (AECOPD).
IVIG Plus Glucocorticoids Effective for Treating COVID-19 Pediatric Syndrome

A large multicenter clinical trial has found intravenous immune globulin (IVIG) plus glucocorticoids may be better than IVIG alone for treating multisystem inflammatory syndrome in children (MIS-C) caused by COVID-19.
Decentralized Clinical Trials: Coming Virtually to a Research Facility Near You

Virtual clinical trials may increasingly replace in-person trials due to their advantages in helping to bring drugs to market.
Breakthroughs in Stem Cell Research

Optimism continues to grow about the use of stem cells in regenerative medicine highlighted by hundreds of successful clinical trials.