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MIPLYFFA, Niemann-Pick Disease

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 the first treatment to get a nod for the condition, the health regulator said on Friday.

STAT · 12h

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.

FierceBiotech · 2d

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,

Business Insider · 1h

FDA Approves Zevra Therapeutics' MIPLYFFA For Treatment Of Niemann-Pick Disease Type C

Zevra Therapeutics Inc. (ZVRA) said that the U.S. Food and Drug Administration approved MIPLYFFA (MY-PLY-FAH) (arimoclomol) capsules

Daily Journal · 15h

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).

Legionnaires' disease, Gaston County

KTLA-TV Los Angeles on MSN · 3d

Health officials in L.A. Co. investigating rare disease spread from raccoons to humans

The Los Angeles County Department of Public Health is looking into two human cases of a rare parasitic infection that comes from raccoons. Baylisascaris procyonis is a type of parasite that lives in the intestines of raccoons;

Yahoo · 12h

Officials have detected a cluster of Legionnaires' disease cases in Gaston County

Gaston officials announced Friday that they are investigating a cluster of confirmed cases of Legionnaires' disease in the county. County spokesman Adam Gaub said the county has confirmed nine cases since May.

The Charlotte Observer on MSN · 13h

9-case cluster of rare pneumonia being investigated by Gaston County health officials

Public health officials are investigating a cluster of nine confirmed cases of Legionnaires’ disease among people who recently visited Gaston County, according to a county spokesperson. Legionnaire’s disease,

13h

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 ...

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 ...

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 ...

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 ...

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 ...

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.

10d

Golf tournament to raise money for research on rare disease

The Little Giants Foundation will hold its first-ever golf tournament to raise money for research into a rare, life-limiting ...

FierceBiotech7d

FDA pushed to be more open, collaborative to accelerate rare disease R&D

The FDA should be more open and collaborative to unleash a surge in approvals of rare disease drugs, according to a report by ...

12hOpinion

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 ...

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