BOSTON, Mass. — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, is pleased to announce the Health Canada approval of MYALEPTA™ (metreleptin for injection). As an adjunct to diet, MYALEPTA is indicated as a...
treatment News
BOSTON, Mass. — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced the publication of results from a Fabry disease patient survey in the peer-reviewed Orphanet Journal of Rare Diseases. “There continues to...
PARMA, Italy — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced presentations at the Society of Inborn Errors of Metabolism (SSIEM) 2026 Annual Symposium, held August 25-28, 2026, in Helsinki, Finland. The...
BOSTON, Mass. — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, announced today that the U.S. Food and Drug Administration (FDA) approved FILSUVEZ® (birch triterpenes) topical gel for the treatment of partial thickness wounds...
COPENHAGEN, Denmark — Ascendis Pharma A/S (Nasdaq: ASND) today announced new radiographic data from Week 104 from the completed pivotal ApproaCH Trial of once-weekly TransCon CNP (navepegritide) in children with achondroplasia. In the trial, TransCon CNP-treated children demonstrated continued improvements in lower extremity alignment through up to two years of...
Gothenburg, Sweden – Children with cystic fibrosis (CF) who were treated with Kaftrio (elexacaftor/tezacaftor/ivacaftor) maintained stable lung function over one year after reducing the frequency of hypertonic saline and other airway-clearance treatments, according to a prospective multicentre study. The reduction in treatment frequency was also associated with fewer days of...
Denver, Colorado – Four children who acquired HIV in utero have remained free of detectable HIV for more than one year, according to new findings that were presented on March 6, at the Conference on Retroviruses and Opportunistic Infections (CROI) in Denver, Colo. The study is part of ongoing, multinational...
Philadelphia, PA – In a landmark study, an international consortium led by researchers at Children’s Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent...
INGELHEIM am RHEIN, Germany – China’s National Medical Products Administration (NMPA) has approved JASCAYD® (nerandomilast) as the first new treatment for adults with progressive pulmonary fibrosis (PPF) in more than five years, marking a breakthrough for millions affected by the life-threatening lung condition.4 The approval, based on the largest clinical trial program in PPF...
PARIS, France – The National Medical Products Administration (NMPA) in China has approved two innovative Sanofi medicines for rare hematologic diseases: Qfitlia (fitusiran) for hemophilia and Cablivi (caplacizumab) for acquired thrombotic thrombocytopenic purpura. These approvals mark another step in Sanofi’s long-term commitment to China, reinforcing the company’s ambition to bring...