Mutations in BRCA1 do not appear to contribute significantly to the risk of prostate cancer progression, according to DNA ...

Stoke Therapeutics Inc. (Nasdaq: STOK) has pulled in Biogen (Nasdaq: BIIB) to help it develop and commercialize a treatment ...

BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

BMRN reports better-than-expected fourth-quarter results, driven by encouraging uptake for Voxzogo. It reiterates its guidance of generating $4 billion in total sales by 2027.

Analysts provide deeper insights through their assessments of 12-month price targets, revealing an average target of $94.25, a high estimate of $126.00, and a low estimate of $70.00. This current ...

Also Read: Sanofi Genzyme Facility Draws FDA Criticism Over Alleged Quality Lapses An independent data monitoring committee ...

Fabry Disease Clinical Trial Pipeline Major pharmaceutical companies are working to advance the pipeline space and unlock the future growth ...

Orchard Therapeutics has tasked Robin Kenselaar with accelerating growth of Lenmeldy, the world’s most expensive drug, ...

Through policy framework shifts, appeals to the court for rare disease funds, and starting a centre in Bengaluru, Prasanna ...

Q4 2024 Earnings Call Transcript February 12, 2025 Operator: Good morning. My name is Jennifer and I will be your conference ...

Seven rheumatology researchers have authored a paper questioning the utility of the 2024 National Academies’ broad definition of long COVID.

Genzyme has fallen foul of the UK pharma industry code, for making misleading and disparaging claims about a competitor product. AstraZeneca has also been named in advertisements along with the ...