In a new discovery, chronic pain has been shown to be physiologically different from acute pain and now scientists have the roadmap for how to target it. While many people with neuromuscular diseases ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

Finding knowledgeable, expert clinicians to treat rare conditions is a challenge in itself, says guest writer Kelly Berger, who offers tips.

Entering the age of 2 years, almost most children will start to actively explore their surroundings. However, there are some who appear lethargic due to muscle weakness in their bodies. This condition ...

Capricor Therapeutics announced that the U.S. FDA has granted Orphan Drug Designation to its lead cell therapy candidate, Deramiocel, for the treatment of Becker Muscular Dystrophy (BMD). This ...

Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, ...