Just a few weeks after Avidity Biosciences made a stir with new data on its experimental therapy for Duchenne muscular dystrophy ... DMD patients with DMD mutations amenable to exon 44 skipping ...

Some of the market’s biggest winners start off as the smallest players. While trillion-dollar tech giants like the ...

Can we fix errors in our genetic blueprint that lead to debilitating disorders like Huntington's disease?How can we better support newcomers to Canada ...

Limb-girdle muscular dystrophy type 2D (LGMD2D/R3) is a rare genetic disorder caused by mutations in the SGCA gene, leading to defective folding and the loss of functional α-sarcoglycan, with ...

Patient advocate Austin Leclaire, who died Feb. 1, played a pivotal role in spurring the development of the first medicines ...

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today reported consolidated financial results for the fourth quarter and full year ended December 31, 2024, and provided ...

Feb. 3, 2025 — Psoriasis is a chronic skin disease that manifests itself mainly with skin symptoms (dryness, itching, scaly skin, abnormal patches and plaques). It affects about 2% of the ...

Their results ... DNA Damage Can Last Unrepaired for Years, Changing Our View of Mutations Jan. 15, 2025 — While most known types of DNA damage are fixed by our cells' in-house DNA repair ...