Phase 3 trial results show levacetylleucine improves SARA scores in patients with the rare disorder ataxia-telangiectasia.

Non-protein-coding genes have been linked to a hereditary condition, retinitis pigmentosa, that causes progressive blindness.

A look at Amyotrophic Lateral Sclerosis (ALS), focusing on motor neuron degeneration, sporadic versus familial ...

PIP is broken down into two parts with daily living starting at £73.90 for the lower rate, rising to £110.40 for higher ...

Larimar Therapeutics is deeply undervalued, trading at ~$3.40 despite a de-risked safety profile and a clear regulatory path ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for myotonic dystrophy type 1 (DM1), the most common adult-onset form of ...

SGT-212 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations - - Dosing of the first participant in the Phase 1b FALCON trial has been completed, with initial data expected ...

Solid Biosciences Inc. (Nasdaq: SLDB) (the "Company” or "Solid”), a life sciences company developing precision genetic ...

Solid Biosciences Inc. (Nasdaq: SLDB) (the "Company" or "Solid"), a life sciences company developing precision genetic medicines for neuromuscular ...

Quince Therapeutics (QNCX) rated Buy ahead of Phase 3 NEAT readout in A-T; eDSP targets $1B+ orphan market with strong cash ...

Brits plagued by forgetfulness are being told to get their vitamin levels checked, as running low on a crucial nutrient can ...

FALCON is a first-in-human, open-label, multi-center Phase 1b clinical trial designed to evaluate the safety and tolerability ...