Rare Disease
This Collaborative Platform Aims to Speed up Rare Disease Drug Development
In a recent webinar hosted by the National Organization for Rare Disorders (NORD), a number of speakers discussed an up and coming project spearheaded by
Rare Patient News. Well Done.
CARE-PWS Results Announced on LV-101 for Prader-Willi Syndrome
- August 11, 2020
Parkinson’s Foundation Awards $1.5M in Grants
ICYMI: SM-88 Receives Orphan Drug Designation as Pancreatic Cancer Treatment
New Co-Pay Relief Program Aids Those with Acromegaly
PODCAST: WAIT, HOW DO YOU SPELL THAT?
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Cerebral Palsy
OWEAR Makes Datasets and Software More Accessible to Aid Research Technology
By Danielle Bradshaw from In The Cloud Copy Shimmer Research has announced that OWEAR – the Open Wearables Initiative – has decided to make their
Acoustic neuroma
Moebius Syndrome Patient Explains How to Emote While Wearing a Mask
Moebius Syndrome Moebius syndrome is a rare condition that causes an inability to move the eyes laterally and facial paralysis. A similar type of paralysis
Methylmalonic Acidemia
New Investigative Therapy for Methylmalonic Acidemia and Propionic Acidemia is Making Regulatory Progress
Fast Track Designation and Rare Pediatric Disease Designation have just been awarded from the FDA to HemoShear Therapeutics. This designation was given to their new
Uveitis
ASRS 2020: Clearside Biomedical Presentations
COVID-19 can’t stop the American Society of Retina Specialists (ASRS)! Although their meeting needed to be held online this year thanks to the viral
Rare Disease
New Study Suggests Targeted Next Generation Sequencing Can Aid in Identification of Menkes Disease
In a recent press release, biopharmaceutical company Fortress Biotech announced that their study regarding ATP7A targeted next generation DNA sequencing for Menkes disease was
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