Margarida Maia, PhD,  science writer—

Margarida is a biochemist (University of Porto, Portugal) with a PhD in biomedical sciences (VIB and KULeuven, Belgium). Her main interest is science communication. She is also passionate about design and the dialogue between art and science.

Articles by Margarida Maia

PROTAC therapy shows promise for SBMA, study finds

ARD-1676, a medication that works by tagging toxic proteins for destruction in cells, promotes clearance of the androgen receptor (AR) protein, which is faulty and clumps in spinal and bulbar muscular atrophy (SBMA), a study found. Earlier approaches to finding a treatment for SBMA have had little success in…

Spinraza can protect adults with SMA from breathing decline

For adults with spinal muscular atrophy (SMA), a genetic disease that progressively weakens breathing muscles, the approved treatment Spinraza (nusinersen) offers a crucial long-term benefit — stable lung function over several years. Data from a multi-center observational study also found that a patient’s initial breathing capacity is the…

FDA denies approval for higher dose of nusinersen for SMA

The U.S. Food and Drug Administration (FDA) has declined to approve a higher dose of nusinersen — the medication in Biogen’s long-approved therapy Spinraza — for the treatment of spinal muscular atrophy (SMA). According to Biogen, no issues were raised with the clinical data in the company’s submission seeking regulatory…

Drug combination improves symptoms in SMA mouse model

An experimental oral combination of two therapies eased motor symptoms and prolonged survival in a mouse model of spinal muscular atrophy (SMA), a study shows. The study, “The effect of coadministration of D156844 and AR42 (REC-2282) on the survival and motor phenotype of mice with spinal muscular atrophy,”…

School-age SMA children more independent on Spinraza: Study

After Spinraza (nusinersen) treatment for slightly longer than one year, school-aged children with spinal muscular atrophy (SMA) showed improved motor function and more independence in tasks involving their arms and hands, a study from China has found. These motor function improvements were linked to reduced symptoms of anxiety…

SMA gene therapy GC101 found safe, effective in animals

Gene therapy GC101 was found to rescue disease manifestations in a mouse model of spinal muscular atrophy (SMA) type 3 and to be safe in non-human primates, supporting its move into clinical testing. Genecradle Therapeutics is sponsoring ongoing Phase 1/2 clinical trials in SMA type 1 (NCT05824169),…