Rare disease
Everything filed under areas in our medical research coverage, the 10 reports filed on it so far.
FDA Launches Rare Disease Framework Initiative
The FDA announced the Rare Disease Framework Initiative on October 14, 2026, to create a tailored regulatory pathway for rare disease treatments...
Shionogi Acquires IntraBio for $2 Billion to Expand Rare Disease Portfolio
Shionogi has agreed to acquire biotech firm IntraBio for $2 billion in cash, gaining the marketed rare disease therapy Aqneursa.
Invivyd chairman Marc Elia becomes CEO
Infectious disease biotech Invivyd has appointed board chairman Marc Elia as its new CEO.
Ionis' Zanvastro Wins First FDA Approval for Alexander Disease
The FDA has approved Ionis Pharmaceuticals' Zanvastro, the first treatment for the rare neurological disorder Alexander disease.
FDA approves Ionis’ zilganersen as Zanvastro for Alexander disease
The FDA has approved zilganersen, marketed as Zanvastro, as the first treatment for Alexander disease, a rare and fatal neurodegenerative disorder.
UniQure Seeks FDA Approval for Huntington's Gene Therapy
UniQure has submitted AMT-130, a gene therapy for Huntington's disease, to the FDA for accelerated approval based on three-year data showing it can...
Alnylam defends silencer drugs after AstraZeneca trial failure
Alnylam defends its RNAi drugs after AstraZeneca's heart disease trial failure, arguing they work even for patients on stabilizer therapies.
Revolution Medicines' Rasonque Approved for Pancreatic Cancer
The FDA has approved Rasonque, a new pancreatic cancer drug by Revolution Medicines, marking the first treatment to target a genetic cause of the...
Capricor’s Duchenne Drug Study Highlights the Challenges of Rare Disease Trials
The FDA's recent decision on Capricor’s deramiocel for Duchenne muscular dystrophy underscores the complexities of rare disease clinical trials...
FDA Clears Bristol Myers Squibb's Oral Multiple Myeloma Therapy, Introducing First-in-Class Agent
The U.S. FDA has approved Bristol Myers Squibb’s oral drug iberdomide, marketed as Zenbexus, for use with daratumumab and dexamethasone in patients...