The company's clinical development plan comprises three global Phase III studies, including two focused on infants and a third for older patients.

Hans Schambe joins BOOST Pharma – a clinical-stage biopharmaceutical company developing novel off-the-shelf cell therapies for rare paediatric skeletal diseases – from Galecto Inc., where as president ...

This time, her baby did test positive for SMA type 1, a severe neurodegenerative genetic condition that typically results in ...

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 ...

Biogen’s successor to Spinraza (nusinersen) has shown benefit in spinal muscular atrophy (SMA) patients who had suboptimal clinical status despite prior administration of gene therapy. In the Phase Ib ...

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

When winter sets in and food becomes scarce, some mammals hibernate, entering a state of deep rest that slashes their energy needs and allows them to fast for months. However, fasting deprives them of ...

Mutations that cause spinal muscular atrophy (SMA) may lead to abnormalities in the development of cells in the spinal cord, a study found.

Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies for spinal muscular atrophy, Duchenne muscular dystrophy and Becker muscular ...

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 ...

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 ...

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