Topic
rare-disease-research
1 stories related to this topic, newest first.
channel4.comworld3 hrs ago
Parents fund custom gene therapy for daughter with ultra-rare mutation
Nadine and David Lipworth have assembled researchers in Australia and the United States to develop an antisense oligonucleotide treatment for their nine-year-old daughter Sasha, who has a spontaneous SLC6A1 mutation. The therapy is in final laboratory testing, with a possible fir…