Alice Melão,  —

Articles by Alice Melão

Device Detects Heart-related Mitochondrial Function in Real Time

Boston Children’s Hospital researchers have developed a device that can detect real-time oxygen levels in mitochondria and predict the effect those levels will have on organs. Their study dealt specifically with predicting heart failure, a common consequence of mitochondrial disease. The team collaborated with Pendar Technologies on the device’s development. Their…

Experts Establish LHON Clinical, Treatment Recommendations

A global panel of leading experts in the field of mitochondrial optic neuropathies has issued a consensus statement to provide clinical and therapeutic guidance for the management of Leber’s hereditary optic neuropathy (LHON). Focusing on Santhera Pharmaceuticals’ Raxone (idebenone), the only treatment approved by the European…

Latest Clinical Data Presented on GS010 for Mitochondria Disease of the Eyes

The latest clinical data continue to demonstrate the therapeutic potential of GenSight Biologics‘ lead candidate GS010 for the treatment of Leber’s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease. These data further support ongoing Phase 3 clinical trials. Trial data were presented at the 25th annual congress of the …

Evaluating Respiratory Enzyme Activity in Kidney Biopsies May Help Diagnose Mitochondrial Disease, Study Finds

Determining the respiratory chain enzyme activity in kidney tissue samples could provide useful information for diagnosing mitochondrial dysfunction in children with renal impairment, according to a report in the Journal of Clinical Medicine. Diseases stemming from mitochondrial dysfunction can be difficult to diagnose because of symptoms similar to other…

Epilepsy Therapies Can Worsen Some Mitochondrial Disorders, Experts Warn

Epilepsy is a common manifestation of mitochondrial disorders for about half of the people with the energy-production malfunction. In most cases, doctors can use epilepsy therapies to treat epilepsy stemming from mitochondrial problems. But the treatments can worsen mitochondrial disorders that are caused by POLG1 gene mutations, according to a review…

Phase 2/3 Trial of Elamipretide to Treat Barth Syndrome Now Enrolling Patients

A Phase 2/3 clinical trial of elamipretide, a potential treatment for a rare mitochondrial disease known as Barth syndrome, is now enrolling patients, the therapy’s developer, Stealth BioTherapeutics, announced. The TAZPOWER study (NCT03098797) will be conducted in McKusick-Nathans Institute of Genetic Medicine, at the Johns Hopkins University School…