Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...

The SMA gene therapy candidate OAV101 IT, a version of Zolgensma, can be safely given to previously treated patients, per new ...

With ongoing breakthroughs in gene and RNA therapies, coupled with regulatory encouragement and AI-based innovation, the market for treating rare neurological diseases is on the cusp of a ...

We recently published a list of 10 Best Short-Term Stocks To Buy Right Now. In this article, we are going to take a look at ...

Roche (RHHBY) announced that it will present new data at the Muscular Dystrophy Association, MDA conference, 16-19 March, 2025, in Dallas, ...

A child with a rare genetic disorder — spinal muscular atrophy type I — was treated prenatally for the first time. Courtesy of Jakob Owens  via Unsplash During an amniocentesis test, a long, thin ...

A multidisciplinary approach, early diagnosis, and comprehensive caregiver education are crucial in effectively managing ...

SMA patients want PROMs that focus on more than physical function, and that also assess mental health, sexual function, sleep ...

The challenge of the obesity epidemic is widespread and one that even pediatricians face constantly,” a Connecticut doctor ...

What is Guillain-Barré syndrome? Guillain-Barré syndrome (GBS) is also called acute inflammatory demyelinating polyradiculoneuropathy (AIDP). It's a neurological disorder in which the body's immune ...

Congress did not reauthorize the rare pediatric disease priority review program at the end of 2024. Advocates say the ripple ...

One of the major drivers propelling the spinal muscular atrophy market is the rising prevalence of rare genetic diseases. These are disorders caused by gene mutations that impact a minuscule ...