Rare Disease Report
Patients & Caregivers


Revance Therapeutics’ lead drug candidate DaxibotulinumtoxinA for Injection (RT002) has been granted orphan drug designation by the FDA to treat cervical dystonia.
Alfred George, Jr., M.D. of Northwestern University summarizes presentations on ATP1A3 in Japan in September
Alynylam has initiated submission of a rolling NDA to the U.S. FDA.
A new study revealed that ATF5 proteins can become implanted in other substances in the brains of patients with Huntington’s disease.
Patisiran, an investigational RNAi therapeutic being developed by Alnylam, has been granted an accelerated assessment by the European Medicines Agency (EMA).
Everybody else is figuring out what they want to do for a job, school, vacations. And I'm barely getting through every single day.
While hearing loss is just 1 of multiple side effects that many risk cancer survivors experience, this loss touches almost every aspect of one’s life.
Is the immune system involved in the progression of HD? And can drugs that target the immune system help HD patients?
Rare Disease Report recently spoke with Jonathan Lim, M.D., the Chairman and CEO of Ignyta about the exciting results and next steps for Entrectinib.
Can rilmenidine delay disease progression in Huntington's disease? Cambridge researchers are trying to find out.
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