English-American guitarist, singer, and songwriter Peter Frampton is best known for his hit album “Frampton Comes Alive!” and his upbeat, immersive performances. But he is also known for his efforts…
Arimoclomol is a treatment that is in development for ALS, Niemann-Pick disease type C (NPC), and inclusion body myositis (IBM). A recent Phase 2/3 trial has ended in failure, unable…
Orphazyme's latest trial has ended in failure after arimoclomol, a possible treatment of inclusion body myositis (IBM), failed to meet any of its endpoints. As the drug has shown massive…
Vance Robinson was diagnosed with inclusion body myositis over ten years ago, and since then he has worked to raise money and awareness. Pre-pandemic, Vance would throw the first pitch…
According to a story from globenewswire.com, the US Food and Drug Administration (FDA) recently accepted the New Drug Application (NDA) for arimoclomol. This application is for the use of the…
Myositis Myositis is an autoimmune disease that leads to muscle damage. Patients live with chronic disability due to the symptoms of this rare condition. These include skin rashes, pain, weakness, fatigue,…
According to a story from globenewswire.com, the biopharmaceutical company Orphazyme A/S has recently announced that it has submitted its New Drug Application (NDA) for its experimental treatment arimoclomol to the…
CytRx Corporation has recently received the Fast Track designation for their ALS treatment, arimoclomol. This status was also given for the treatment of Niemann-Pick disease and sporadic Inclusion Body Myositis…
Lisa Christopher-Stine is the Director of the Myositis Center at Johns Hopkins. She recently shared her biggest tips for patient care and some updates from the field, hoping to change…
Music and rock 'n' roll lovers already know him name, but Peter Frampton soon will become a new type of rock star: rare disease advocate. The British guitar legend announced…
Niemann-Pick Disease is a genetic lysosomal storage disease. Lipids accumulate in the lysosomes of cells, causing debilitating symptoms. There are three forms of the disease- A, B, and C. Each…
According to a story from globenewswire.com, the biopharmaceutical company Orphazyme has officially endorsed a set of guidelines that outline how rare disease patient advocacy organizations and drug companies should interact…
According to a story from GlobeNewsWire, the biopharmaceutical company Orphazyme A/S, which is committed to the development of treatments for patients that are living with rare diseases, has recently announced…
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