Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral ...

Novartis has significantly strengthened its neuroscience pipeline over recent years, topped by the $12bn acquisition of ...

Axial myopathy is a general term for a group of myopathies affecting the axial muscles. Axial myopathy appears to be a grossly underestimated feature of many muscle diseases and often overlooked ...

An analysis of genetic data from over 900,000 people shows that certain stretches of DNA, made up of short sequences repeated ...

As of Thursday, January 08, Dyne Therapeutics, Inc.’s DYN share price has dipped by 5.03%, which has investors questioning if ...

A large genetic study shows that many people carry DNA sequences that slowly expand as they get older. Common genetic ...

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced business and program updates ahead of upcoming investor meetings in January, including the company’s scheduled webcast from the 44th ...

LoQus23 Therapeutics Ltd ("LoQus23"), a biotechnology company investigating small molecule drugs that could stop the pathogenic triplet expansion that is the cause and driver of Huntington's Disease ...

Company on track to report ELEVATE-44-201 data from the first cohort in Q2 2026 and ELEVATE-45-201 data from the first cohort in mid-2026 –-- ...

An analysis of genetic data from over 900,000 people shows that certain stretches of DNA, made up of short sequences repeated ...

There is a genetic feature known as a repeat expansion, in which short sequences are repeated continuously. Some of them ...

LoQus23 Therapeutics, based at Granta Park in Cambridge, has stepped up its bid to combat Huntington’s Disease (HD). It has ...