A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would ...

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people.

A mother of two terminally ill children said that Hilary Swank ‘took the time to learn’ about her family after their ...

Jada Bafus told Page Six that she and the actress quickly came to realize it was a case of “two triggered mothers” having a ...

After learning devastating news about our young son, we needed to feel connected to friends and family, not just exist beyond ...

To continue the effort to raise enough money to help the family of Darren McNutt acquire a specialized van to make it easier ...

We are advised by Ractigen Therapeutics that journalists and other readers should disregard the news release, Ractigen Therapeutics Doses First Patient in IIT Study of RAG-18 for Duchenne Muscular ...

Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced the successful dosing of the first patient in its investigator-initiated trial (IIT) of ...

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