Jonathan Strober, MD, explains the initial presentation of myasthenia gravis in a pediatric patient population and why initial diagnosis for these patients can be challenging.

DMD treatment SAT-3247 was safe and well-tolerated, and showed signs of increasing muscle strength in five adults, Satellos ...

Hopkins neuroscientist Richard Huganir is close to finding a potential life-changing treatment for kids with SYNGAP1-related ...

"The newer medications that are coming out, and a lot of them are approved, are much more focused on the problem," explains ...

It's a slow-moving train wreck," Mike Graglia says about his 12-year-old son Tony's rare genetic disease with no cure.Caused ...

In April, the Deparment of Health and Human Services shuttered the committe that oversaw newborn screenings. Advocates hope it can be reinstated.

Modalis Therapeutics Corporation (Tokyo Stock Exchange: 4883), a pioneering company developing innovative products for the treatment of rare genetic diseases utilizing its proprietary CRISPR-GNDM® ...

Some patients with a rare form of ALS benefited from an experimental therapy, with biomarker evidence of reduced injury to neurons and even limited functional recovery.

When Columbia neurologist and scientist Neil Shneider speaks to his ALS patients who volunteer for experimental therapies, he ...

Tilray Brands, a leader in the cannabis industry, recently announced a proposal to execute a reverse stock split. It hopes ...

ZYUS Life Sciences Corporation (the "Company") , a Canadian-based life sciences company focused on the development and commercialization of novel cannabinoid-based pharmaceutical drug candidates for ...

Inozyme recently featured new clinical insights and hosted an educational symposium at the Joint Congress of the European ...