Patients & Caregivers
Aegerion Pleads Guilty to Marketing Orphan Drug Juxtapid Improperly
Juxtapid was approved by the FDA in December 2012 for the treatment of homozygous familial hypercholesterolemia (HoFH).
Pompe Combination Therapy Gets Orphan Designation
Chaperone drug AT2221 binds to ATB200 to help stabilize the latter ERT until it reaches its target (lysosomes).
Avrobio Developing a Gene Therapy for Pompe Disease
It is the third gene therapy the company has in its pipeline to treat a rare lysosomal disease (in addition to Fabry and Gaucher therapies)
Dup15q Mom Stresses the Importance of the Internet
In this video, Dup15q mom Sarah Rasumussen stresses the importance of using the internet when working with a rare disease.
De Novo Mutations
New study found that fathers pass along about 4 times as many new mutations as mothers do.
NDA Filing for Promising RNAi Therapy for Amyloidosis Expected Soon
The Phase 3 clinical trial testing patisiran to treat ATTR amyloidosis with polyneuropathy has met its primary endpoint.
4D Molecular Receives $3 Million from Cystic Fibrosis Foundation Therapeutics
One year after its initial grant of $525,000, Cystic Fibrosis Foundation Therapeutics has granted $3 million in additional funding to 4D Molecular Therapeutics.
Ongoing Study of PBI-4050 in Alstrom Syndrome
Prometic Life Sciences has released data from an ongoing Phase 2 open-label study, highlighting the safety and efficacy of PBI-4050 in patients with Alström Syndrome.
Familial Adenomatous Polyposis Treatment Gets FDA Fast Track Status
The U.S. Food and Drug Administration granted “Fast-Track” status to CPP-1X/sul for the treatment of adults with familial adenomatous polyposis this morning.
Ask the Experts: Dr. Jaya Trivedi Talks Periodic Paralysis
Rare Disease Report spoke with Jaya Trivedi, MD, professor of neurology at UT Southwestern Medical Center in Dallas, Texas, about periodic paralysis.
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