EMA backs conditional EU nod for Vijoice in rare life-threatening overgrowth disorder PROS

The company is expected to conduct further studies to confirm the long-term efficacy and safety of Vijoice in both adult and paediatric patients

MicrobioTx launches IBS Risk Score to enable early detection of gut disorder

The launch coincides with IBS Awareness Month in April

Glycomine completes enrollment in Phase 2b trial for rare genetic disorder

The study is a global, randomized, double-blind, placebo-controlled trial testing GLM101 for phosphomannomutase 2 congenital disorder of glycosylation

University of Leeds launches landmark HALO study on lifelong impact of childhood blood disorders

Backed by a £680,000 grant from Leeds Hospitals Charity, the study is jointly led by academics and clinicians from the University of Leeds and Leeds Teaching Hospitals NHS Trust

FDA nod to first treatment for rare genetic brain disorder

The approval comes after a systematic review of published literature

FDA okays JUXTAPID for kids with ultra-rare genetic cholesterol disorder

EU to withdraw Levamisole medicines over risk of rare, deadly brain disorder

The recommendation is based on new data from continuous safety monitoring of medicines in the EU

Mirum Pharmaceuticals bags Canadian nod for LIVMARLI to treat rare liver disorder

LIVMARLI’s new tablet form offers patients weighing 22 kg or more a convenient, one-tablet-per-dose option, complementing the existing 9.5 mg/ml oral solution

AGC Biologics powers commercial launch of its life-changing gene therapy for rare childhood immune disorder

Waskyra is an ex vivo gene therapy that uses patients’ own CD34+ hematopoietic stem and progenitor cells, genetically engineered with a lentiviral vector

Nia Therapeutics unveils first implantable brain interface for memory disorders

The SNS records neural activity from 60 channels across four brain regions—far exceeding current commercial devices