by Madaline Spencer | Mar 27, 2025
A recent study illustrated clinical improvements in patients with retinal dystrophy treated with gene therapy. Retinal dystrophy is a rare ophthalmology condition caused by a genetic deficiency of AIPL1. The condition is characterized by severe and rapid progression...
by Madaline Spencer | Mar 26, 2025
Takayuki Shirakawa, PhD, Research Scientist at Mitsubishi Tanabe Pharma, discusses the effects of LRSAM1 on TDP-43 in patients with amyotrophic lateral sclerosis (ALS). ALS, also referred to as “Lou Gehrig’s disease,” is a motor neuron disease...
by Madaline Spencer | Mar 25, 2025
The U.S. Food and Drug Administration has approved Fabhalta (iptacopan) for the treatment of C3 glomerulopathy (C3G) in adults. C3G is a rare kidney disease characterized by damage to kidney glomeruli due to abnormal activation of the complement system. When C3...
by Madaline Spencer | Mar 25, 2025
Savara Therapeutics has announced a new diagnostic test for autoimmune pulmonary alveolar proteinosis (aPAP). aPAP is a rare autoimmune lung disorder. It is the most common form of pulmonary alveolar proteinosis. There is a wide variance in disease...
by Madaline Spencer | Mar 24, 2025
The U.S. Food and Drug Administration has approved Amvuttra (vutrisiran) for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) in adults. ATTR-CM is a rare amyloidosis caused by aging or genetic mutations. This results in the misfolding of TTR protein...
