People frequently wait years to be "taken seriously," postcode lotteries for access to care and a general lack of knowledge are among some of the key findings from a University of Aberdeen ...

Please provide your email address to receive an email when new articles are posted on . Regeneron Pharmaceuticals Inc. and Intellia Therapeutics Inc. have announced an expanded research collaboration ...

The degree to which novel value elements such as insurance value impact estimated treatment value for rare, severe genetic diseases such as Duchenne muscular dystrophy is unclear. Objectives: To ...

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

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 ...

Atamyo Therapeutics, a biotechnology company specializing in the development of next-generation gene therapies for limb-girdle muscular dystrophy (LGMD), announced at the MDA (Muscular Dystrophy ...

Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are caused by mutations in the DMD gene, which reduces the amount of dystrophin, a protein that protects muscle fibers. DMD ...

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 ...

If cancer is a disease of overabundance, where cells divide without restraint and tumors grow despite the body's best interests, then degenerative diseases are disorders of deprivation. When ...