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 ...

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 ...

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

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 ...

It isn't hard to see what makes Maine Coons such widely beloved cats all over the world. With their rugged builds, ...

After all, with each passing year, more and more pet parents seek out Maine Coon breeders and speciality cat rescues to claim ...

Clinical & Scientific Conference, researchers and clinicians are talking about the central role of patients, families, and caregivers in driving change for people living with neuromuscular diseases.