GeneDx Holdings WGS announced a sponsored genetic testing program with Zevra Therapeutics to improve diagnosis for patients suspected of having Niemann-Pick Disease Type C (NPC), a rare and ...

A 4-year-old Las Vegas boy is battling Niemann-Pick Type C, a fatal disease affecting just 4 children in Nevada. His family is fighting insurance barriers, traveling cross-country for care.

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) ...

LEIDEN, Netherlands--(BUSINESS WIRE)--Azafaros has announced that data from the ongoing double-blind, placebo-controlled Phase 2 RAINBOW study investigating its lead asset, nizubaglustat in patients ...

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 ...

(RTTNews) - Zevra Therapeutics Inc. (ZVRA), a rare disease therapeutics company, Thursday, announced the commercial availability of Miplyffa for the treatment of Niemann-Pick disease type C along with ...

Zevra Therapeutics ZVRA shares are up on Tuesday following a new partnership with GeneDx Holdings Corp. WGS to enhance genetic testing for Niemann-Pick Disease type C, or NPC. Niemann-Pick disease ...

Following the recent recommendation of its advisory committee, the U.S. FDA gave the nod to Zevra Therapeutics Inc.’s arimoclomol, a day ahead of its Sept. 21 PDUFA date, as the first treatment for ...

Barbara Lazarus’ two sons, David and Daniel, were healthy and active kids, but things changed in their early 20s. David developed severe psychiatric symptoms, which were diagnosed as schizoaffective ...

Patients with Niemann-Pick disease type C who received N-acetyl-L-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, showed reduced neurologic signs and ...

The recommended dosage is based on the patient’s body weight. The Food and Drug Administration (FDA) has approved Aqneursa ™ (levacetylleucine) for the treatment of neurological manifestations of ...

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 ...