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.
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.
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.
