Amino acid balance could play a role in SMA, as a study showed acids able to influence nervous system function were ...

Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 ...

The FDA has accepted for Priority Review the BLA for apitegromab for the improvement of motor function in patients with spinal muscular atrophy.

A Prescription Drug User Fee Act (PDUFA) target action date for apitegromab has been set for Sept. 22, 2025.

Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating ...

We recently published a list of the 7 Most Undervalued Biotech Stocks To Invest In. In this article, we are going to take a ...

Novartis' OAV101 IT showed a 2.39-point HFMSE improvement in SMA patients vs. 0.51 points in the sham control group. The STRENGTH study found OAV101 IT stabilized motor function in patients who ...

Medication costs are only a piece of the puzzle when clinicians think about health care access, explains Leigh Maria Ramos-Platt, MD. Addressing the cost of gene therapies has remained a prominent ...

BOSTON--(BUSINESS WIRE)--Curadel Pharma, a pioneer in zwitterionicity and innovator in advanced radiotherapies and drugs for optical surgical navigation, announced today that it has initiated the ...

PacBio (NASDAQ: PACB), a leading provider of high-quality, highly accurate sequencing platforms, today announced a newly published study in Nature Communications unveiling a powerful new method for ...

Read our disclosure page to find out how can you help Windows Report sustain the editorial team. Read more If you’re trying to find Advanced System Settings in Windows 10, you’re in the right place.

A UConn professor and researcher who 28 years ago discovered a gene for a protein that limits human muscle growth is closer than ever to having that work play a significant role in treating those with ...