The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However,…
Articles by Patricia Inácio, PhD
Exenatide, a drug that mimics the effects of the gut hormone GLP-1, increased the levels of frataxin — a protein…
A natural genetic mechanism may help reverse the genetic defect within the FXN gene — excessive repeats of a portion of…
A Phase 1 trial evaluating Chondrial Therapeutic’s investigational therapy CTI-1601 as a treatment for Friedreich’s ataxia patients has…
Seelos Therapeutics received a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) covering the parenteral, or non-oral…
Friedreich’s ataxia can occur as a late-onset disease, with symptoms appearing after age 50, a case-report shows. Late-onset cases lack…
Seelos Therapeutics received a Notice of Allowance from the United States Patent and Trademark Office covering the use…
The oral glucose tolerance test showed higher sensitivity, compared with other diagnostic tools, and was able to detect up to three…
Takeda and StrideBio have established a collaboration to develop new, more effective adeno-associated viruses (AAV) to be used…
A large, European registry study shows that Friedreich’s ataxia patients have a considerable spectrum of clinical symptoms that go beyond…