• Latest Posts

Sensorion receives positive opinion from EMA for treatment of hearing loss

Ayala Pharmaceuticals presents interim data on desmoid tumor trial

SiSaf reveals positive preclinical data in siRNA program to treat osteopetrosis

Ionis presents positive study results at International Symposium on amyloidosis

Avacta’s drug targeting soft tissue sarcoma granted Orphan Drug Designation by FDA

Sanofi’s first treatment in the U.S. approved for adults and children with ASMD

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Emactuzumab designated as an orphan medicinal product for TGCT in Europe

Amgen announces top-line results from biosimilar phase 3 study

Ocelot Bio receives FDA Orphan Drug Designation for lead candidate for treatment of hepatorenal syndrome

Vaderis emerges from stealth to start HTT trial

Emtora to use $16.9M grant to develop treatment for rare genetic disease

Aeglea BioTherapeutics’ arginine 1 deficiency treatment gets EMA ok

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