Ultragenyx bags FDA priority review for groundbreaking GSDIa gene therapy

If approved, DTX401 would be the first treatment to address the disease at its root cause

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

Ionis and Otsuka score EU nod for DAWNZERA, offering new hope for rare HAE patients

The EU approval of DAWNZERA is an important milestone that reflects our ongoing commitment to bring this innovative medicine to people in need across the globe

FDA nod to first-ever treatment for fatal pediatric Menkes disease

Ipsen’s FALKON trial falls short in rare FOP study, offers key insights

FOP is a rare genetic disorder caused by mutations in the ALK2 kinase

Bayer advances fight against rare kidney disease with new experimental drug

Alport Syndrome is caused by genetic mutations affecting type IV collagen in the kidneys, ears, and eyes

EU approves Alexion’s Koselugo to treat adults with neurofibromatosis type 1 tumours

Approval based on pivotal KOMET Phase III trial demonstrating significant tumour reduction in adults with NF1

Bartronics India acquires 15% stake in Huwel Life Sciences for Rs. 50 Cr

BIL also holds an option to increase its equity stake by an additional 15%

Dr. Jitendra Singh inaugurates 'National Biobank' at CSIR-IGIB

CSIR-IGIB advances indigenous CRISPR Trials on sickle cell anaemia, anti-microbial resistance, liver fibrosis and rare disorders

Dr. Prabhavathi. R joins HEJJE

Dr. Prabhavathi brings over 14 years of unparalleled experience in pediatrics and a specialized focus on neurodevelopmental disorders