New Phase 1b data support the safety and effectiveness of salanersen over one year in children with SMA who had the potential for improvement due to suboptimal clinical status with prior gene ...

Union Minister H D Kumaraswamy has assured assistance for a two-year-old child suffering from a rare genetic disorder after ...

Joining host Kent Small on The Smallzy Show, Leigh-Anne spoke about how the band, which Jesy quit in 2020, are 'always there ...

US biotech major Biogen has presented additional results from the Phase Ib study of salanersen, an investigational novel ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

Biogen has launched a trio of Phase 3 trials to test salanersen, a new SMA treatment that aims to be a more convenient ...

Newborn screening for SMA enables significantly earlier initiation of disease-modifying treatments. Early treatment was associated with motor function and development in infants with SMA. Prenatal ...

Jesy Nelson has shared another sweet video of her twin girls Ocean Jade and Story Monroe, showing their sweet bond.

More early-stage data show the drug, called salanersen, appeared to have positive effects on neurodegeneration and motor function in patients previously treated with Novartis' Zolgensma.

Jesy Nelson's Little Mix bandmate, Leigh-Anne Pinnock, has opened up about Jesy's twins' diagnosis. Earlier this year, Jesy took to Instagram to announce her twin girls, Ocean Jade and Story Monroe ...

In Trumbull County, a local nonprofit will once again be hiding colorful candy-filled eggs in yards this Easter to help a ...