treatment News

A foreign drugmaker has thrown a gauntlet to the Korean rare disease treatment market. Italy-based Recordati is the company. On Rare Disease Day Feb. 28, the company launched Recordati Korea, its Korean offshoot and Asia-Pacific headquarters, declaring its official advance to the Korean and regional market. Unlike most other pharma...
EMERYVILLE, Calif. — 4D Molecular Therapeutics, a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases in ophthalmology and pulmonology, today announced that the U.S. Food and Drug Administration (FDA) has granted the RMAT designation for the investigational genetic medicine...
EMERYVILLE, Calif. — 4D Molecular Therapeutics, a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for the Company’s product candidate aerosolized 4D-710...
Washington DC – As of Dec. 22, the FDA had approved at least 88 novel treatments in 2023, for indications ranging from breast and colorectal cancer to Duchenne muscular dystrophy to sickle cell disease. Rare diseases such as Rett syndrome and Friedreich’s ataxia saw their first approvals. Some decisions, like Sarepta’s...
Toruń, Poland – Boys suffering from Barth syndrome, a genetic determined disease with a very high mortality rate, finally have hope for a cure. What has contributed to this finding is research of Dr. habil Karolina Mikulska-Ruminska, Nicolaus Copernicus University (Torun, Poland) CU Professor from the Institute of Physics. The...
Two biotechs are shoring up their pipelines and commercial aspirations, tacking on a clinical-stage rare disease drug and a preclinical cancer med, respectively. Fortress Biotech-founded Avenue Therapeutics is adding on a phase 1/2-stage asset from AnnJi Pharmaceutical for the treatment of spinal and bulbar muscular atrophy (SBMA). Financial details are...
Auckland, New Zealand – A molecule created at the University of Auckland is one step closer to becoming a treatment for an extremely rare and severely debilitating neurological disorder called Phelan-McDermid syndrome. According to Neuren Pharmaceuticals, a biopharmaceutical company that is listed on the Australian Securities Exchange, children with the...
The future treatment of Parkinson’s Disease has undergone tremendous development in recent years. Now, a breakthrough in research has emerged, delivering the strongest results for both side-effect-free and long-lasting treatment effects.   Aarhus, Denmark – The results were just published in Nature Communications under the title “Enhanced production of mesencephalic...