
Opinion|Videos|September 4, 2024
Promising Therapy Approaches for Advanced Friedreich Ataxia
A key opinion leader explores new and promising treatment approaches for managing advanced stages of Friedreich ataxia.
Advertisement
Episodes in this series

Video content above is prompted by the following:
- In your opinion, what are the most promising emerging therapeutic avenues for managing advanced stages of FA, and how do you see them impacting clinical practice in the future?
Advertisement
Related to this article

Insmed launches once-daily brensocatib in Japan, the country's only approved treatment for noncystic fibrosis bronchiectasis.

Cross-ancestry GWAS of more than 1.8 million people flags SERPINA1, SMAD3, and other bronchiectasis targets shared with COPD and chronic bronchitis.

The FDA approved bepirovirsen, the first antisense oligonucleotide for chronic hepatitis B, offering a new treatment with functional cure potential.

ALIS added to azithromycin and ethambutol raised culture conversion in new MAC lung disease, with rare resistance, in an ENCORE analysis at IDWeek 2026.

Insmed’s once-daily TPIP prodrug shows sustained lung vasodilation in PAH and PH-ILD, improving walk distance and other key measures.

Cosibelimab received an NCCN Category 2A preferred designation for locally advanced CSCC, supported by durable responses and low immune toxicity.

ASPEN links green sputum to neutrophils and exacerbations; brensocatib (DPP1) lowers purulence, while meta-analysis supports fewer flare-ups in bronchiectasis.
Advertisement
Advertisement
Trending on AJMC
1
Bepirovirsen Approval Offers New Option for Chronic HBV Treatment
2
Inhaled Amikacin Raises Culture Conversion in New MAC Lung Disease
3
Bronchiectasis GWAS Highlights Candidate Targets Shared With COPD
4
Uninsured Rate Rises to 11.2% as Coverage Erodes Before Medicaid Cuts
5


