Spinal Muscular Atrophy
Zolgensma for Spinal Muscular Atrophy Now Approved in Japan
In May of 2019, the FDA approved a new therapy for Spinal Muscular Atrophy (SMA) called Zolgensma. Now, that therapy has been approved by the
Rare Patient News. Well Done.
Review of Rare Movement Disorders in India and the United States
- March 30, 2020
New Phenylketonuria Treatment Granted Orphan Drug Designation
Experimental Treatment for Cryopyrin-Associated Periodic Syndrome (CAPS) Shows Potential in Early Trial
Immunocompromised During COVID-19? Not Much Has Changed in the Lives of Many Rare Patients
LATEST PATIENT WORTHY ARTICLES
Lupus
Despite Advancements in Precision Medicine, We Also Need to Look at Disease Broadly
Published in Social Science & Medicine, a recent study conducted by Drexel University has shed light on how, even in a world of evolving precision medicine,
Rare Disease
Chloroquine was Touted to Treat Coronavirus. Now, This Sjögren’s Syndrome Patient Can’t Find It
According to a story from Buzzfeed News, Sharon Greenstein, who lives with the rare autoimmune disease known as Sjögren’s syndrome, can no longer find the
Antiphospholipid Syndrome
No Adverse Reactions between Abortion and Autoimmune Rheumatic Diseases
Is it safe for those with autoimmune rheumatic diseases to undergo the termination of a pregnancy? According to research published in Arthritis & Rheumatology, the
Rare Disease
Editor’s Choice: A Few More Covid-19 Articles
Hi everyone, We know you might be overwhelemed with virus news– and next week, we’ll switch the theme up. For now, we wanted to send
Rare Disease
New Research Discovers Childhood Neurodegenerative Disorder
Researchers from the Murdoch Children’s Research Institute (MCRI) have recently discovered a previously unknown and uncharted neurodegenerative disorder. This rare disorder, which has not yet
