Muhle is one of many supporters of Legislative Bill 338 from State Sen. Dave “Woody” Wordekemper of Fremont, which seeks to ...

Based on China's accumulated rare disease knowledge and genetic data from its population, PUMCH-GENESIS is the world's first ...

Based on China's accumulated rare disease knowledge and genetic data from its population, PUMCH-GENESIS is the world's first ...

A Phase III trial evaluating nex-z in transthyretin amyloidosis with cardiomyopathy is underway, with plans for another ...

Ionis relies on upfront payments and licensing fees from partners, as well as Spinraza royalties, to drive revenue today, but its rapidly advancing portfolio should help diversify revenue. 2025 is ...

Boehringer Ingelheim, IP Group, the UK Respiratory Gene Therapy Consortium (GTC) 1 and OXB, 1 today announce the start of LENTICLAIR TM 1, a Phase I/II trial of BI 3720931, a novel, first-in-class, ...

Spinal muscle atrophyorspinal muscular atrophy(SMA) is a genetic disorder that can affect babies, children, and adults. A ...

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that it will present additional data from the pivotal Phase 3 ...

We thank Michèl Willemsen and colleagues for their interest in the ATTeST trial of intra-erythrocyte dexamethasone sodium phosphate in children with ataxia telangiectasia.1 The two doses studied in ...

Art Still has ATTR amyloidosis, an inherited heart condition that dramatically impacts Black men and the former Kansas City ...

Management outlined significant revenue contributions from TRYNGOLZA and donidalorsen in 2025. TRYNGOLZA is positioned to increase patient identification and diagnosis over time, with a gradual launch ...

The former Offaly hurling manager features in this week's Laochra Gael episode and opens up on his ongoing health battle ...