Business Wire

Niemann-Pick Disease | A Pipeline Analysis Report 2018 | Technavio

LONDON--(BUSINESS WIRE)--Technavio has announced their latest pipeline analysis report on the Niemann-Pick disease market. The report includes a detailed analysis of the pipeline molecules under ...

Zevra Therapeutics Executes Distribution Agreement to Broaden Access to MIPLYFFA® for the Treatment of Niemann-Pick Disease Type C (NPC)

NPC is an ultra-rare, relentlessly progressive, genetic disorder that leads to premature mortality. MIPLYFFA, used in conjunction with miglustat, is the only treatment shown to halt disease ...
Business Wire

International Niemann-Pick Disease Registry and Mandos Health announce new research collaboration using real-world data to evaluate the treatment effects of VTS-270 to manage ...

TYNE AND WEAR, England--(BUSINESS WIRE)--The International Niemann-Pick Disease Registry (INPDR) and biopharmaceutical company Mandos Health today announce a new collaboration giving Mandos Health ...
The American Journal of Managed Care

Study on Niemann-Pick Disease Yields New Insights, Novel Variants

A total of 92 gene variants were sequenced, 41 of which were novel. A new report offers novel insights into links between the clinical, biochemical, and genetic characteristics of Niemann-Pick disease ...
Healio

FDA approves second Niemann-Pick disease type C treatment

Please provide your email address to receive an email when new articles are posted on . The FDA has approved Aqneursa as an oral treatment for neurological symptoms associated with Niemann-Pick ...
Monthly Prescribing Reference

Miplyffa Approved for Niemann-Pick Disease Type C

Findings showed that among patients who also received miglustat, treatment with arimoclomol slowed NPC disease progression. The Food and Drug Administration (FDA) has approved Miplyffa ™ (arimoclomol) ...
Austin American-Statesman

Austin's IntraBio gets FDA approval for Niemann-Pick Type C1 treatment. What to know.

Six years ago, Austin sisters Belle and Abby Andrews were diagnosed with Niemann-Pick Type C1, a rare genetic disorder sometimes called childhood Alzheimer’s. Belle, who was 6 at the time, was falling ...
STAT

My son’s rare disease was renamed ASMD. Why are people still calling it Niemann-Pick?

Ayik is an advocate for people living with rare diseases. Recently, the smoldering embers of conflict in my family’s rare disease community have burst into flames over the use of an eponym: ...
Local 12 WKRC Cincinnati

8-year-old diagnosed with rare disease that gives her childhood dementia

CHESTERFIELD, Va. (CNN Newsource/WTVR/WKRC) - A Disney song helps take 8-year-old Abby Alvey to another world, a world where she's free of the stress and issues related to her terminal diagnosis of ...
ABC15 Arizona

Valley baby showing great progress with experimental Niemann-Pick Type C disease drug treatments

A Valley 2-year-old who is fighting a rare and fatal disease is now showing improvement with an experimental drug. Woodrow Miller suffers from Niemann-Pick Type C disease, described as a form of ...
AZ Central

An experimental drug gave hope to children with a rare disease. Now, families fear it will be taken away

As an infant, Parker Miller's skin turned yellow and his belly swelled. His liver was so damaged doctors feared he would need a transplant. Woodrow Miller was born seven weeks premature, only 4 pounds ...
Healio

FDA approves Miplyffa for Niemann-Pick disease type C

Please provide your email address to receive an email when new articles are posted on . The FDA announced in a press release that it has approved Miplyffa to treat neurological symptoms associated ...