BioSpace56m
Alnylam’s Amvuttra Approved as First RNAi Silencer for Rare Type of Cardiomyopathy
The FDA approval of Alnylam’s Amvuttra sets up a three-way race with Pfizer and BridgeBio, which both market transthyretin ...
US FDA expands approval for Alnylam's drug to treat rare heart disease
U.S. Food and Drug Administration approved Alnylam's drug to treat a rare and deadly heart disease on Thursday, allowing the ...
WTAP1h
Researchers believe they may have found a way to delay Alzheimer’s
An experimental treatment appears to delay Alzheimer’s symptoms in some people genetically destined to get the disease in ...
US FDA approves Alnylam's drug for rare heart condition
U.S. Food and Drug Administration approved Alnylam's drug to treat a rare and deadly heart disease on Thursday, allowing the ...
Managed Healthcare Executive4h
FDA Approves First-Ever Cell Therapy Treatment for Rare Progressive Eye Disease
Encelto is small, semi-permeable capsule implanted in the eye that contains allogeneic retinal pigment epithelium cells ...
The Atlanta Journal-Constitution5h
Experimental treatment appears to delay Alzheimer’s symptoms but funding at risk
An experimental treatment appears to delay Alzheimer’s symptoms in some people genetically destined to get the disease in their 40s or 50s, according to new findings from ongoing research now caught ...
The Lancet5h
The artist and the scientist join forces for rare diseases
Art and science are disciplines that have historically co-existed as polarised entities. However, artists and scientists both ...
WKMS8h
Rare bat species found at Mammoth Cave helping in the fight against White Nose Syndrome
While some bat species have seen up to a 99% population decline, Rafinesque's big-eared bat has shown a strong resistance to the fungal disease.
Pharmaceutical Commerce10h
PAP 2025: The Impact of Out-of-Pocket Costs on Patients with Rare Diseases
Jonathan James, CEO, Hope Charities, discusses the critical importance of maintaining access to life-saving medications for ...
The Lancet15h
Advancing policies for rare diseases in Europe
Fostering dialogue between neurologists, researchers, patient representatives, and policymakers is an important step towards ...