Published: May 01, 2021
Expanded indications for patients with sickle cell disease or other anemias, as well as thalassemia
BOSTON, May 1, 2021 /PRNewswire/ Chiesi Global Rare Diseases, a business unit of
Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), announced today that the U.S. Food & Drug Administration (FDA) has approved FERRIPROX
® (deferiprone) for the treatment of transfusional iron overload due to sickle cell disease (SCD) or other anemias in adult and pediatric patients 3 years of age and older. This FDA approval expands the use of FERRIPROX for patients with SCD or other anemias as well as patients with thalassemia regardless of prior iron chelation exposure.
LogicBio Announces Presentation of Retrospective Study of Disease Course in Pediatric Patients with Severe Methylmalonic Acidemia
- Data presented at ACMG meeting evaluate disease progression in non-transplanted patients and children receiving liver transplantation
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LEXINGTON, Mass., May 3, 2021 /PRNewswire/ LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood, today announced results from a retrospective study of the disease course in pediatric patients living with severe methylmalonic acidemia (MMA) caused by a deficiency of the mitochondrial enzyme methylmalonyl-CoA mutase (MMUT). The study results were recently highlighted in a poster presentation at the American College of Medical Genetic and Genomics (ACMG) Annual Clinical Genetics Meeting.
/PRNewswire/ Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group),.
Press release content from Globe Newswire. The AP news staff was not involved in its creation.
Timber Pharmaceuticals Receives Decision to Grant Japanese Patent for Lead Asset TMB-001
Timber PharmaceuticalsApril 28, 2021 GMT
- Company is evaluating a topical pharmaceutical composition of isotretinoin for the treatment of moderate to severe subtypes of congenital ichthyosis -
Basking Ridge, NJ, April 28, 2021 (GLOBE NEWSWIRE) via NewMediaWire Timber Pharmaceuticals, Inc. (“Timber” or the “Company”) (NYSE American: TMBR), a biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases, today announced that the Japanese Patent Office has decided to grant a patent (No. 2018-542677) for its lead asset, TMB-001, the Company’s topical pharmaceutical composition of isotretinoin that is currently being evaluated for the treatment of moderate to severe subtypes of congenital ichthyosis (CI).
Protalix BioTherapeutics and Chiesi Global Rare Diseases Receive Complete Response Letter for Pegunigalsidase Alfa from FDA
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CARMIEL, Israel and BOSTON, April 28, 2021 /PRNewswire/ Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx
® plant cell-based protein expression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), today announced that they received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the Biologics License Application (BLA) seeking accelerated approval of pegunigalsidase alfa (PRX‑102) for the proposed treatment of adult patients with Fabry disease.