Patients & Caregivers
RT002 Granted FDA Orphan Drug Designation
Revance Therapeutics’ lead drug candidate DaxibotulinumtoxinA for Injection (RT002) has been granted orphan drug designation by the FDA to treat cervical dystonia.
Report from the 6th ATP1A3 Meeting; Tokyo, Japan
Alfred George, Jr., M.D. of Northwestern University summarizes presentations on ATP1A3 in Japan in September
Alnylam Initiates Submission of NDA for hATTR Treatment
Alynylam has initiated submission of a rolling NDA to the U.S. FDA.
Spanish Study Reveals Role of ATF5 Protein in HD
A new study revealed that ATF5 proteins can become implanted in other substances in the brains of patients with Huntington’s disease.
EMA Grants Alnylam Accelerated Assessment for Potential hATTR Amyloidosis Treatment
Patisiran, an investigational RNAi therapeutic being developed by Alnylam, has been granted an accelerated assessment by the European Medicines Agency (EMA).
Being Afraid to Dream: Life with Chemotherapy-Induced Hearing Loss
Everybody else is figuring out what they want to do for a job, school, vacations. And I'm barely getting through every single day.
A Parent's Silent Prayer: Preventing Hearing Loss for Childhood Cancer Survivors
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.
Can Manipulating the Immune System Help Patients with Huntington's Disease?
Is the immune system involved in the progression of HD? And can drugs that target the immune system help HD patients?
Ignyta Chairman and CEO: Entrectinib Has A Really Good Shot
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 a Blood Pressure Medication Help HD Patients?
Can rilmenidine delay disease progression in Huntington's disease? Cambridge researchers are trying to find out.
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