Dottie Caplan, who previously served as Senior Vice President, Patient Advocacy and Engagement, will be responsible for leading Applied’s patient engagement and advocacy efforts as well as the ...

Data shared by the company showed that galactosemia incidence is about 3,300 patients in the U.S., with 80-100 new births per year, and 4,400 patients in the E.U., with 120 new births per year.

Galactosemia is a rare genetic, metabolic disorder caused by a deficiency in the galactose-1-phosphate uridyltransferase (GALT). This means patients cannot break down galactose, causing a buildup of ...

“Included na po sa Z benefit package ng PhilHealth the 10 rare diseases po, including Maple Syrup Urine Disease , Methylmalonic Acidemia or Propionic Acidemia , Galactosemia , Phenylketonuria, yang ...

The biotech could draw on the support of the Galactosemia Foundation as it tries to win over the FDA. ... Investors sent Applied’s share price down 80% to $1.75 when the news broke late Wednesday.

The NDA included data from the phase 1/2 ACTION-Galactosemia study (ClinicalTrial.gov Identifier: NCT04117711) and the phase 3 ACTION-Galactosemia Kids trial (ClinicalTrial.gov Identifier ...

The US Food and Drug Administration (FDA) has declined to approve Applied Therapeutics’ govorestat for classic galactosemia, prolonging the wait for the first marketed treatment for the genetic ...

Additional supportive studies resulted in robust efficacy and safety data across 185 patients with Classic Galactosemia over 3 years. The results of the ACTION-Galactosemia Kids study and the Phase ...

NEW YORK, Nov. 27, 2024 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a biopharmaceutical company dedicated to creating transformative treatments for rare disease, today announced ...

NEW YORK - Applied Therapeutics Inc . (NASDAQ:APLT), a biopharmaceutical company, received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its New Drug ...