Rare Diseases – Talk Bio

TriNetX Achieves Record as the Most Cited Real-World Electronic Health Record Dataset in Peer-Reviewed Research

1 month ago talkbio0Tagged Citation, Clinical Trials, Data Analytics, Electronic Health Records, global network, Medical Research activity, Peer-Reviewed Research, Rare Diseases, real-world data, TriNetX

TriNetX; real-world data; electronic health records; peer-reviewed research; citations; medical research; data analytics; global network; rare disease; clinical trials

Researchers Publish Roadmap of FDA Interactions After Baby KJ’s Groundbreaking Gene Editing Therapy, as FDA Signals New Pathway for Patient-Specific Treatments

1 month ago talkbio0Tagged approval pathway, Baby KJ, CPS1 deficiency, CRISPR gene editing, Disease Gene Therapies, Infrequent, N-of-1 therapies, platform trials, Precision Medicine, Rare Diseases, regulatory roadmap, United States Food and Drug Administration

Baby KJ; CRISPR gene editing; CPS1 deficiency; FDA approval pathway; personalized medicine; rare disease; regulatory roadmap; platform trials; N-of-1 therapies; rare disease gene therapies

Unlocking Japan’s Potential in Rare Disease Drug Development: Recent Progress and Challenges (2025)

1 month ago talkbio0Tagged Clinical Trials, drug lag, Drugs, Orphan, innovation, Japan, pharmaceutical partnerships, Rare Diseases, regulatory approval

rare disease; Japan; orphan drug; drug lag; innovation; regulatory approval; pharmaceutical partnerships; clinical trials

Alkermes Acquires Avadel Pharmaceuticals for $2.1B, Gaining Sleep Drug LUMRYZ and Setting Strategic Evolution

2 months ago talkbio0Tagged Acquisition, Alkermes, Avadel Pharmaceuticals, Biological Factors, LUMRYZ, Narcolepsy, portfolio, premium, Rare Diseases, Sleep medicine specialist, strategic

Alkermes; Avadel Pharmaceuticals; acquisition; LUMRYZ; narcolepsy; sleep medicine; strategic portfolio; biopharmaceuticals; rare disease; deal premium

Sanofi’s efdoralprin alfa (INBRX-101) achieves all endpoints in Phase 2 alpha-1 antitrypsin deficiency (AATD) trial

2 months ago talkbio0Tagged Acquisition, alpha 1-Antitrypsin Deficiency, efdoralprin alfa, INBRX-101, Inhibrx, Phase 2, plasma-derived therapy, Rare Diseases, sanofi, Zemaira

Sanofi; efdoralprin alfa; INBRX-101; AATD; alpha-1 antitrypsin deficiency; Phase 2; Inhibrx; acquisition; rare disease; plasma-derived therapy; Zemaira

Novo throws weight behind budding rare disease pipeline

2 months ago talkbio0Tagged complement system, MASP-3 Inhibitor, Novo Nordisk, Omeros, Paroxysmal nocturnal hemoglobinuria, Payments, pipeline, Rare Diseases, Zaltenibart

Novo Nordisk; rare disease; pipeline; Omeros; Zaltenibart; MASP-3 inhibitor; PNH; complement system; milestone payments

Gene Therapy Delivers Long-Term Immune Protection in Children With Rare Immune Disorders

2 months ago talkbio0Tagged Adenosine deaminase deficiency, Clinical Trials, Combined immunodeficiency disease, Congenital leukocyte adherence deficiency, Curative Therapy, gene therapy, immune protection, Leukocyte adhesion deficiency type 1, Long-term, Pediatric immunology, Rare Diseases, Severe (severity modifier)

gene therapy; long-term immune protection; ADA-SCID; severe combined immunodeficiency; LAD-I; leukocyte adhesion deficiency; rare disease; pediatric immunology; clinical trial; curative treatment

BioCryst Snaps Up Fellow Angioedema Player Astria for $700M

2 months ago talkbio0Tagged Acquisition, Angioedemas, Hereditary, Astria, BioCryst, Cash, Drug Industry, navenibart, Rare Diseases

BioCryst; Astria Therapeutics; acquisition; hereditary angioedema (HAE); navenibart; rare disease; pharmaceutical industry; cash and stock deal; Jill Milne; $700 million