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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 · 8h

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 · 10h

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

MarketWatch · 11h

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,

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;

The Charlotte Observer on MSN · 8h

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,

Yahoo · 11h

Cluster of Legionnaires’ disease cases confirmed in Gaston County, source unknown

The Gaston County Public Health Department is investigating a cluster of Legionnaires’ disease cases confirmed in the area. Health officials said the confirmed cases are among people who lived, or have spent time,

New York Post9h

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 ...

7hOpinion

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 ...

WEWS Cleveland, OH on MSN5h

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 ...

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.

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 ...

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 ...

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