Aravindhan Veerapandiyan, MD, pediatric neurologist at Arkansas Children’s Hospital, discusses the AFFINITY clinical trial testing the safety and efficacy of RGX-202 for patients with Duchenne muscular dystrophy (DMD).
DMD is a rare disease affecting the muscles, leading to muscle wasting that gets worse over time. It is caused by genetic changes in the DMD gene. This gene is responsible for encoding dystrophin, a protein involved in muscle cell structure and signaling pathways. DMD occurs primarily in males, though in rare cases may affect females. The most common symptom of DMD is progressive weakness and loss (atrophy) of both skeletal and heart muscle. Early signs of the disease may include delayed ability to sit, stand, or walk and difficulties learning to speak.
RGX-202 is a gene therapy being tested to produce a shorter version of naturally occurring dystrophin and its C-Terminal domain element. The treatment is designed to support delivery and targeted expression of genes in the heart and skeletal muscles with a NAV AAV8 vector.
AFFINITY is a phase 1/2 clinical trial testing the safety and efficacy of RGX-202 in patients with DMD. Increased expression of RGX-202 microdystrophin and a decrease from baseline in serum creatinine kinase (CK) levels have been observed in all enrolled patients. The treatment has also shown a well tolerable profile with no serious adverse events. For more information, visit https://clinicaltrials.gov/study/NCT05693142
To learn more about rare neurological diseases, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/
