Two Scots parents are pleading with the NHS to allow their "fighter" son access to a life changing drug before it is too late ...

Sam Millar, eight, was diagnosed with Duchenne muscular dystrophy with his parents told there was a life-changing drug that ...

The FDA has approved Soliris the first-ever treatment for children with generalized myasthenia gravis gMG offering new hope in pediatric neuromuscular care Learn more!

CT should become the fifth state to add Duchenne to the screening newborns receive, giving parents the early answers they ...

Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...

Santhera Pharmaceuticals Holding AG (OTCMKTS:SPHDF – Get Free Report) saw a significant growth in short interest during the month of February. As of February 28th, there was short interest totalling 3 ...

PTC Therapeutics, Inc. (NASDAQ:PTCT – Get Free Report)’s share price hit a new 52-week high during mid-day trading on Friday after JPMorgan Chase & Co. raised their price target on the stock from $72.

Adolescence is far more grown-up than its name might suggest. The four-part series, starring Stephen Graham, Ashley Walters ( ...

Explore the Genome Editing Market size, share, trends, and future growth. Learn about CRISPR, TALENs, ZFNs, and advancements shaping genetic research & medicine.

St. Anthony’s Memorial Hospital already offers many pediatric therapy services to care for children with diagnoses such as ...

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

The Manchester Road Race Committee announced that it will be donating $100,250 from the proceeds of last years Thanksgiving day run to organizations in the ...