Life-saving treatments for children with orphan illnesses often move slowly, if at all, through the federal approval process.
The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...
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NTRA debuts Zenith Genomics next-gen sequencing test for rare diseases
Natera NTRA announced the commercial launch of Zenith genomics, a next-generation whole genome sequencing assay designed to ...
Although FDA Commissioner Marty Makary promised “an exciting treatment” for autism, what the agency delivered was a label expansion for leucovorin to treat the ultrarare cerebral folate deficiency.
Together, NORD and OpenEvidence aim to strengthen the connection between clinical knowledge and patient education, helping ...
In Israel, approximately 50,000 children live with rare genetic diseases and syndromes, and thousands more are diagnosed each year. Nedirim, a non-profit organization founded by parents of children ...
Torie Bosch is the First Opinion editor at STAT. Celena Lozano’s son Benny, who turned 5 in November, loves trains, trucks — anything that goes. He also has a rare disease, PURA syndrome. Earlier this ...
Registration has opened for the online "Run for Kids with Rare Diseases" charity race, allowing participants to raise funds ...
The Alliance was born from a classic D.C. moment: three Illinois advocates, standing in a hallway, waiting to meet with Senator Dick Durbin. That is where Wayne Galasek of Oak Park, Doug Butchart of ...
Federal health officials on Monday laid out a proposal to spur development of customized treatments for patients with hard-to ...
The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO's investigational gene therapy, ...
Leigh syndrome is a rare metabolic disorder that begins in childhood and causes severe neurological and muscular problems.
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