Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies ...

Biogen’s salanersen is now heading into three Phase III trials in SMA.

SMA mutations disrupt spinal cord development, with fewer nerve cells in early stages. SMN protein deficiency leads to a mesodermal fate bias, with fewer motor neurons. Increasing SOX2 pathway ...

It consisted of 18 women participating in a 5-day dry immersion test to assess the risk of developing potentially life-threatening blood clots. The results support existing evidence that women are at ...

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

STELLAR-2, a randomized, double-blind, sham-controlled study, will evaluate the effects of salanersen when initiated ~6 months after onasemnogene abeparvovec-xioi in infants with SMA who received ...

Biotechnology firm Scholar Rock is poised to make a significant strategic shift from a development-stage company to a commercial entity. The catalyst for this t ...

Barclays 28th Annual Global Healthcare Conference March 12, 2026 8:30 AM EDTCompany ParticipantsDavid Hallal - ...

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 ...

Leigh‑Anne Pinnock has spoken warmly about Jesy Nelson, calling her former Little Mix bandmate “incredible” for the way she has handled her baby twins’ recent health diagnosis. Jesy, who welcomed ...

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 ...