Disease

Neuraxpharm and Minoryx win EU marketing authorisation for NEZGLYAL, the first oral drug approved for cerebral adrenoleukodystrophy in young boys.

Firefly Neuroscience will introduce NeuroSigma's Monarch eTNS sleep-worn neurostimulator to its US clinician network as a non-drug ADHD option for children.

A Washington University cohort study used Alamar's 130-protein plasma panel to show amyloid clearance and cognitive response reflect distinct biological processes.

Five-year CARTITUDE-2 data show 50% of RRMM patients remained alive and progression-free after a single cilta-cel infusion, with overall survival at

Pharming's sNDA for lower leniolisib doses in children weighing 13-27 kg with APDS has a PDUFA date of 30 January 2027.

Five-year CARTITUDE-2 data show half of early-line RRMM patients remained alive and progression-free after a single cilta-cel infusion, with no

aTyr Pharma has agreed a Phase 3 protocol with the FDA for efzofitimod in pulmonary sarcoidosis, with study activities due to begin in Q4 2026.

MediciNova has finished the double-blind portion of its 234-patient Phase 2b/3 ALS trial and expects topline results by end of 2026.

The Cambridge biotech's in vivo Prime Editor targets the root genetic cause of alpha-1 antitrypsin deficiency, with first-in-human data expected in

Seven of eight enrolled participants met the primary endpoint in the upLIFT Phase 3 study; topline results are expected before end of 2026.

Satellos Bioscience has cleared an IND for its AAK1 inhibitor in a second muscle disease, backed by US$5m from the FSHD Canada Foundation.

The London biotech has enrolled over 100 patients in a trial testing S-pindolol benzoate to preserve lean mass during and after semaglutide therapy.

Actinogen Medical's phase 2b/3 XanaMIA trial has completed all 247 patient visits, with topline results for its oral cortisol inhibitor expected in November.

Adaptyx Biosciences has secured FDA Breakthrough Device Designation and TAP entry for its continuous cortisol wearable, targeting Cushing syndrome and adrenal

Lirafugratinib becomes a second-line option for FGFR2-rearranged cholangiocarcinoma after posting a 46% confirmed response rate in the ReFocus trial.

PhAST Corp.'s Gram-negative panel delivers antimicrobial susceptibility results in under 2.5 hours directly from positive blood cultures, cutting a typical 24-48

Interim IMAgINATION trial data show sefaxersen significantly reduced proteinuria versus placebo at 37 weeks in 459 adults with IgAN.

SOTIO Biotech has secured Fast Track and Orphan Drug designations from the FDA for SOT106, its LRRC15-targeting ADC, ahead of a planned first-in

Both EMBARQ-CSU studies hit primary and key secondary endpoints, with complete response rates above 40% at 12 weeks; a BLA filing is planned for

The Italian medtech closed a €15m Series A and implanted its Silkothane® hybrid arteriovenous graft in its first patient in an ongoing Italian pilot trial.