US FDA approves Zevra's treatment for rare genetic disease
The U.S. Food and Drug Administration has approved Zevra Therapeutics' drug for a rare and fatal genetic disorder, making it ...
MedPage Today8h
Women With MS Less Likely to Receive Treatment Than Men
Women had a lower probability of receiving any disease-modifying treatment (DMT) and were even less likely to get ...
US FDA approves Elanco's skin disease treatment for dogs
The U.S. Food and Drug Administration has approved Elanco Animal Health's skin disease treatment for dogs, the health ...
KIMT1d
Scientists highlight lack of facility space to cure diseases
Researchers are on a mission to grow innovative technology to cure diseases and they say facility space could help the ...
Calves to Cure helps fund research for Montana boy's rare disease
The Billings Livestock Commission hosted the sixth annual Calves to Cure to help raise money and awareness for Duchenne ...
Nature5d
Doctors cured her sickle-cell disease. So why is she still in pain?
Gene and cell therapies bring fresh hope to people with genetic disorders, but recovery can be complex and long-term support ...
Science Daily1d
New therapy that targets and destroys tau tangles is a promising future Alzheimer's disease treatment
Scientists have developed new potential therapies that selectively remove aggregated tau proteins, which are associated with Alzheimer's disease, and improve symptoms of neurodegeneration in mice.
Zevra Therapeutics’ MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment for Niemann-Pick Disease Type C
MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive ...
Fatty Liver Disease: Risk Factors & Treatment Options
Fatty liver disease happens when fat builds up in your liver. This can cause damage, inflammation, and other complications.
New biomarkers can help target treatment for chronic skin disease sarcoidosis
Sarcoidosis is a rare systemic disease that afflicts an estimated 200,000 people in the U.S., more often people who are Black ...
Rocket: Danon Disease Treatment Program Should Carry The Tide Until Regulatory Moves
Rocket Pharmaceuticals announced enrollment completion for phase 2 study using its gene therapy RP-A501 for treatment of male ...
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Calves to Cure helps fund research for Worden boy's rare disease
The Billings Livestock Commission hosted the sixth annual Calves to Cure on Thursday, an event to help raise money and ...