The potential for cell and gene therapies is growing, offering groundbreaking treatments for rare genetic disorders and ...

FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration ...

The NIH is considering a catastrophic cut to indirect cost reimbursements for public universities, which would hurt the ...

Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 weeks compared with sham in patients aged 2 to 18 years with spinal muscular ...

Funding medical research is about more than just numbers. It's real people,” says Ryan Manriquez, president of the UC ...

Imagine the joy of holding your newborn for the first time, only to hear doctors say your baby would live to the age of two.

Here is our list of the 11 Best Gene Therapy Stocks to Buy According to Analysts.

Gene therapy companies have faced numerous setbacks over the last few years. What will it take for gene therapies to fulfill ...

Natco Pharma updates on Risdiplam launch in India, Delhi High Court denies Roche's injunction plea, pricing set at ₹15,900.

In response to a request from the European Medicines Agency (EMA) following the death of a 16-year-old patient in the U.S., ...

Market OverviewThe global Newborn Screening Market is valued at USD 3.44 Billion in 2024 and is projected to reach a value of ...