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Perth Family Confronts Metachromatic Leukodystrophy Diagnosis in Newborn
by Liam Croy A Perth family devastated by a rare genetic disorder has been thrown a lifeline by a world-leading research program in Italy.… Continue Reading
Tunnel Vision, Headaches Lead to Rare Diagnosis of Paroxysmal Nocturnal Hemoglobinuria
by Skyler Hrabe I will never forget the feeling of utter helplessness, despair and disbelief as the realization dawned on me that my… Continue Reading
First Duchenne Muscular Dystrophy Boy in Canada to Begin Drisapersen Redosing
Five years ago, 12-year-old Alex Chiabai was one of the first boy in Canada to begin taking drisapersen, a promising drug that provided a… Continue Reading
The Atlantic: What If (Almost) Every Gene Affects (Almost) Everything?
By Ed Yong (SOURCE) In 1999, a group of scientists scoured the genomes of around 150 pairs of siblings in an attempt to find genes that are… Continue Reading
Project Sleep Announces Winners of 2017 Jack & Julie Narcolepsy Scholarship
Project Sleep is proud to announce the 11 recipients of the 2017 Jack & Julie Narcolepsy Scholarship, a national scholarship program… Continue Reading
RARECast: Summit Pursues A Different Approach to Treating Duchenne
  Duchenne Muscular Dystrophy, a rare genetic disease, causes progressive muscle wasting that slowly robs people of their abilities and… Continue Reading
Rare Disease Report: RASRx is the Latest Duchenne Drug To Get Orphan Status From the FDA
Thanks to Rare Disease Report for this piece. University of Southern California (USC) start-up RASRx hopes to develop a drug to treat… Continue Reading
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