Archive for clinical trials
A new start for gene therapy for ‘bubble boy’ disease: First U.S.-treated patient doing well
Until this month, Agustín Cáceres’s baptism was the only time his family could come close to him. Everyone had to wear masks, gloves and gowns. After that, he went into isolation, along with his mother Marcela, who came out only for meals. His father Alberto, and his four-year-old brother Jeremías, kept to a separate bedroom. ... Read More about A new start for gene therapy for ‘bubble boy’ disease: First U.S.-treated patient doing well
Moving gene therapy into high gear
Gene therapy, still experimental but beginning to enter the clinic, attempts to utilize advanced molecular methods to treat and even reverse genetic diseases. The field started in earnest about 25 years ago and has had many setbacks along the way to its recent earliest successes. International collaboration has been critical. Children’s Hospital Boston is one ... Read More about Moving gene therapy into high gear
With algae blooms hope for a long-acting local anesthetic
For decades, Chile’s shoreline has had problems with periodic algal blooms – referred to as Red Tide, but actually containing a mix of microorganisms including bluegreen algae. Their toxins accumulate in shellfish, landing seafood consumers in the hospital, partially paralyzed, sometimes needing ventilators to breathe. The nerve block caused by the toxins is reversible, so ... Read More about With algae blooms hope for a long-acting local anesthetic
Neurogenetic disorders: Dreaming the impossible dream
People with autism and most other disorders of brain development have never had medications to treat their core behavioral and cognitive symptoms. The best they can get are drugs targeting secondary problems, like irritability or aggression. But now, a new wave of clinical trials aims to change this. In the last decade, scientists have discovered ... Read More about Neurogenetic disorders: Dreaming the impossible dream