The first patient with small cell lung cancer has been dosed in a trial of peluntamig (PT217) combined with chemotherapy.

Scientists in the UK have successfully used gene therapy to restore some vision to legally blind children with an inherited retinal condition. All 11 children in the clinical trial saw improvements ...

PrimeC has been given an ‘Orphan Drug’ status by the FDA (Food and Drug Administration) and EMA (European Medicines Agency).

Pivotal Phase 2b clinical trial (ELPIS II) evaluating Lomecel-B (laromestrocel) in Hypoplastic Left Heart Syndrome (HLHS), a rare pediatric disease and orphan-designated indication, has achieved more ...

Argenx had cash, cash equivalents, and other short-term investments totaling $3.4 billion as of the end of 2024, making it well-positioned to continue funding its pipeline and commercialization ...

Investorideas , a global news source and expert investing resource, announces today's roundup of stocks added to the biotech stock directory.

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, ...

NICE publishes final draft guidance recommending efanesoctocog alfa as an option for treating and preventing bleeding in ...

ORLANDO, FLORIDA / ACCESS Newswire / February 28, 2025 / RedChip Companies will air interviews with Calidi Biotherapeutics, Inc. (NYSE American:CLDI) and OS Therapies Inc. (NYSE American:OSTX) on the ...

Vilobelimab is being developed for various debilitating or life-threatening inflammatory indications, including a Phase 3 trial for pyoderma gangrenosum (PG). Vilobelimab has been granted orphan drug ...

As we observe Rare Disease Day today, let us take a look at seven drugs poised to be approved to address rare diseases in ...