Government of India has provided tax relief on life-saving drugs and medicines used for rare diseases. As per the Ministry of Finance’s response in Rajya Sabha on February 11, 2025, basic customs duty ...

Novo Nordisk announced plans for a longer phase III trial testing its investigational obesity drug CagriSema, with regulatory submission expected early next year. The FDA approved ranibizumab (Susvimo ...

For a long time, lysosomes were considered as mere waste bags for cellular constituents. Thankfully, studies carried out in the past 15 years were brimming with elegant and crucial breakthroughs in ...

Autosomal recessive osteopetrosis was diagnosed in patient 3 at 2.5 months of age, at which time hepatosplenomegaly and optic atrophy were noted. He survives following a matched related BMT with ...

Tracey Dones with her then-newborn son Anthony, who was diagnosed with osteopetrosis four months after he was born. Tracey Dones "It was cool to compare the breakdown with me and my twin brothers," ...

Background Osteopetrosis is a rare, inherited, bone disorder, characterized by osteosclerosis, obliteration of the medullary cavity and calcified cartilage. The autosomal dominant form is ...