News – SMA News Today The Web's Daily Resource for Spinal Muscular Atrophy News
- Challenges of Life as Teen, Young Adult With SMA Highlighted in Surveyby Marisa Wexler MS on March 5, 2021
Being an adolescent or young adult with spinal muscular atrophy (SMA) presents unique challenges, according to a study based on viewpoints expressed by patients ages 12 to 25. “‘I have SMA, SMA doesn’t have me’: a qualitative snapshot […]
- CHMP Favors EU Approval of Evrysdi as 1st Oral, At-home SMA Treatmentby Marta Figueiredo on March 3, 2021
Evrysdi (risdiplam) has been recommended for approval in the European Union (EU) to treat spinal muscular atrophy (SMA) patients, ages 2 months and older, with a clinical diagnosis of type 1, 2, or 3, or with one to four copies of the SMN2 […]
- Evrysdi Seen to Improve Survival, Motor Development of Infants with SMA Type 1by Marta Figueiredo on March 1, 2021
Genentech’s Evrysdi (risdiplam) improved survival in infants with spinal muscular atrophy (SMA) type 1, and allowed them to achieve developmental milestones not usually seen in the natural course of the disease, according to one-year data from the […]
- Mother Raises 4 Adopted Girls From China With SMA, Chronic Illnessesby Hawken miller on February 26, 2021
When Josylnn Jones McLaughlin moved to Guangzhou, China, to teach conversational English in 2012, she didn’t expect to bring home two girls, one with spinal muscular atrophy (SMA) and the other with spina bifida, when she returned to Anderson, South […]
- Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashionby Hawken miller on February 25, 2021
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed on […]
- Cure SMA Offers Free Virtual Therapy Sessionsby Mary Chapman on February 24, 2021
To help with stress or anxiety brought on by the COVID-19 pandemic or other issues, adult patients with spinal muscular atrophy (SMA) in the U.S. are being offered up to three half-hour live video sessions with a licensed therapist. The Adults with SMA […]
- Rare Disease Day at NIH, Set for March 1, Growing Year by Yearby Mary Chapman on February 22, 2021
Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for […]
- Brain Involvement in SMA Type 1 Still Poorly Understood, Review Findsby Marta Figueiredo on February 22, 2021
Children with spinal muscular atrophy (SMA) type 1 may have neurodegeneration in several areas of the brain, with impaired cognitive function and profound speech difficulties, according to a review study. However, brain involvement in this severe type of SMA […]
- Study Finds Varying Perspectives From Parents on Spinraza Therapy for Childrenby Steve Bryson PhD on February 19, 2021
Parents of children with spinal muscular atrophy (SMA) reported different perspectives and concerns about Spinraza (nusinersen) therapy in an interview-based study, done to further understand the decision-making process involved in choosing whether to try the […]
- New SMN-boosting Molecule Shows Promise as Add-on Therapyby Marta Figueiredo on February 17, 2021
Targeting a specific region of an intermediate molecule generated from the SMN2 gene increased the levels of functional SMN — the protein lacking in spinal muscular atrophy (SMA) — in cells derived from SMA patients, a study shows. In […]
