Miplyffa, Niemann-Pick Disease
US FDA approves Zevra's treatment for rare genetic disease
The U.S. Food and Drug Administration approved Zevra Therapeutics' drug for a rare and fatal genetic disorder, making it the first treatment to get a nod for the condition, the health regulator said on Friday.
Zevra Therapeutics' Drug Becomes First FDA-Approved Treatment For Ultra-Rare Neurodegenerative Disease
The FDA has approved Zevra Therapeutics' Miplyffa (arimoclomol) as the first treatment for neurological symptoms of Neimann-Pick disease, type C. This approval follows favorable voting by the Genetic Metabolic Diseases Advisory Committee.
FDA approves first drug for rare neuron disease, Niemann-Pick type C
Parents pushed for years for treatments for the neuron disease Niemann-Pick type C. After setbacks, one drug now has FDA approval.
FDA scraps adcomm for Applied's rare disease drug ahead of November decision
After pushing back the decision date for Applied Therapeutics’ metabolic disorder drug govorestat, the FDA has now decided that a planned advisory committee meeting won’t be required. | After pushing back the decision date for Applied Therapeutics’ metabolic disorder drug govorestat,
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
FDA Approves First Treatment for Niemann-Pick Disease, Type C
Today, the U.S. Food and Drug Administration approved Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC). Miplyffa, in combination with the enzyme inhibitor miglustat,
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 ...
Buy Rating Justified by Azitra Inc’s Advances in Rare Skin Disease Treatments and Financial Stability
Jason McCarthy, an analyst from Maxim Group, reiterated the Buy rating on Azitra Inc (AZTR – Research Report). The associated price ...
Bored Panda on MSN1d
Dog Has A Rare Disease That Makes Her Body Frozen, Surprises Everyone And Recovers Against All Odds
For example, this one vet chose to care for this pitbull puppy despite the fact that she had a rare and very serious disease ...
STAT1d
House panel passes pediatric rare disease bill, setting it up for government funding bill
A House committee on Wednesday passed a bill to renew a program aimed at aiding development of drugs for rare diseases in ...
Rocky River family share story of daughter's rare disease
An Oscar-nominated actor recently spoke exclusively to PEOPLE about his adult son with a rare disease. It inspired a Rocky ...
Woman with rare disease claims she’s now blind and bruised after doctors made her get 3 vaccines
A 23-year-old Florida woman has been temporarily blinded and bruised after allegedly being required to receive multiple ...
Targeted Oncology2d
Atypical Chronic Myeloid Leukemia: A Rare Disease With Poor Prognosis
Ongoing research is exploring new molecular targets and targeted therapies for this challenging disease.
Psychology Today2d
How I Survived a Rare Autoimmune Disease
I walked out of my marriage and immediately fell ill. I was ill for two years. At first, I thought it was a cold that would ...
Commentary: Congress has just weeks to help children with rare diseases
A rare-disease federal incentive program critical to biotech R&D is in jeopardy, writes Leslie Williams, president and CEO of ...