The FDA has granted a rare pediatric disease designation to GPS for the treatment of pediatric patients with AML.1 "GPS has already demonstrated promise in clinical settings for AML, and we believe its potential could extend to pediatric patients,” said Angelos Stergiou, MD, ScD hc, president...
Sarepta provides grant funding for programs that enable education, address unmet needs of rare disease patients, promote the highest standards of care, raise awareness of ongoing research, or focus on other critical community initiatives. Requests will be considered across the spectrum of neuromuscular...
FDA grants rare pediatric disease designation for Leber congenital amaurosis gene therapyYoung, AlexOcular Surgery News
The FDA granted rare pediatric disease designation for BPM31510T for the treatment of epidermolysis bullosa.Image: Adobe Stock. “We are honored to offer hope for children who suffer from debilitating wounds and pain,”Vijay Modur, MD, PhD,chief medical officer of BPGbio...
within tumor HI, there remains a significant unmet need for new therapies directed at hypoglycemia treatment. Ersodetug has shown real-world benefit in patients with insulinoma and preclinical studies have shown that ersodetug can similarly blunt IGF-2 and insulin-mediated insulin-receptor signaling....
Food and Drug Administration (FDA), for the treatment of esophageal cancer. The FDA grants Orphan Drug Designation to treatments for rare diseases in the United States that affect fewer than 200,000 patients. Orphan Drug Designation offers policy benefits to drug developers, including as aid with...
patients with Duchenne - a rare and devastating disease - who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping will now have available the first treatment targeted specifically for this disease subtype," said Billy Dunn, M.D., acting...
Additionally, in a preclinical design, optimized DNAI1 mRNA reduced immune reactivity and was well-tolerated in patients.2 “PCD is a rare genetic lung disorder that currently has no FDA-approved treatments; therefore, receiving this designation is an important recognition for the PCD patient...
PTC Therapeutics is committed to helping the lives of patients and families within our communities. Part of this commitment includes providing grants to support high quality programs that are focused on rare diseases and to make meaningful contributions globally. All grant requests will be submitted to...
surgery that creates a two-ventricle series circulation with the left ventricle (LV) pumping blood to the body and the right ventricle pumping blood to the lungs is the ideal anatomic repair. Unfortunately, achievement of this objective is limited by the inability in most patients for ...