Mirum Pharma Pill Lands FDA Approval for Ultra-Rare Bone Growth Disorder
đź“° Curated Industry Article This is a summary of a full article from a trusted healthcare and insurance publication. Click “Read Full Article” below to read the complete story. Mirum Pharmaceuticals’ Atebrioz is the third FDA-approved drug for fibrodysplasia ossificans progressiva (FOP), but it brings advantages over the two other products for this rare disease […]
Gene Silencing-Biotech ADARx Makes Some Noise With $535M Stock Market Debut
đź“° Curated Industry Article This is a summary of a full article from a trusted healthcare and insurance publication. Click “Read Full Article” below to read the complete story. ADARx Pharmaceuticals upsized its IPO and raised additional cash through a private placement with partner AbbVie. Lead ADARx program onvuzosiran is in Phase 3 testing for […]
New Roche Drug Shows Powerful Results for Kidney Disease
đź“° Curated Industry Article This is a summary of a full article from a trusted healthcare and insurance publication. Click “Read Full Article” below to read the complete story. Roche reported the drug sefaxersen achieved statistically significant and clinically meaningful results in a Phase 3 test in immunoglobulin A nephropathy, or IgAN. This antisense oligonucleotide, […]
Proven Ultragenyx Therapy Is A Life Changing Sanfilippo Fix
đź“° Curated Industry Article This is a summary of a full article from a trusted healthcare and insurance publication. Click “Read Full Article” below to read the complete story. Ultragenyx Pharmaceutical’s Fayuvi is now the first FDA-approved therapy for the underlying cause of Sanfilippo syndrome type A, an ultra-rare inherited neurological disorder. It’s the second […]
Scholar Rock Secures Proven FDA Approval for Rare Disease Drug
đź“° Curated Industry Article This is a summary of a full article from a trusted healthcare and insurance publication. Click “Read Full Article” below to read the complete story. Scholar Rock’s Isembyld isn’t the only FDA-approved therapy for spinal muscular atrophy, but it is the first one that directly targets muscle tissue. Isembyld is the […]