With an infrastructure in place to market its lone commercial product Ruconest, it made sense in 2019 for Pharming Group to scoop up another rare disease candidate in the same arena. Four years later, Pharming’s $20 million purchase of leniolisib from Novartis is set to bear dividends. On Friday, the...
treatment News
New York, NY. Women with atrial fibrillation (AF) undergoing a procedure called pulsed field ablation (PFA) have just as good outcomes as men with AF undergoing the same procedure, according to a large-scale international study led by the Icahn School of Medicine at Mount Sinai. This study is the first...
Shangai, China and TEL AVIV, Israel and ATHENS, Greece and ZURICH, Switzerland — The World Orphan Drug Alliance (WODA), a global alliance of commercial distributors dedicated to providing access to treatments for rare diseases and specialty medicines in complex markets around the world, has expanded its reach to include the...
The Cayman Islands joins the United Nations in observing the first annual Sickle Cell Disease World Day on Friday, 19 June. The worldwide event will raise awareness of the disease, and follows a United Nations General Assembly resolution adopted in December 2008 that recognizes sickle cell disease as a public...
Melbourne, Australia – Researchers at St Vincent’s Institute of Medical Research (SVI) in Melbourne have shown that a commonly prescribed rheumatoid arthritis drug can suppress the progression of type 1 diabetes. The world-first human trial, published today in the New England Journal of Medicine and led by SVI’s Professor Thomas...
Full-service contract research organisation (CRO) Worldwide Clinical Trials has partnered with Every Cure to expedite the discovery of treatments for patients with rare diseases. Worldwide will serve as a drug development and clinical trial partner to Every Cure, a non-profit organisation that aims to ‘unlock the full potential of existing...
BOSTON, Mass. — X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that the U.S. Food and Drug Administration (FDA) has approved XOLREMDI™ (mavorixafor) capsules for use in patients 12 years of age and older with WHIM...
CHICAGO – Xentria Inc., a biopharmaceutical company focused on developing novel biologics to address unmet clinical needs, today announced that the U.S. Food and Drug Administration (“FDA”) has confirmed that the Company satisfactorily addressed all requirements and can proceed with initiation of the planned Phase 1 clinical trial of XTMAB-16....
STOCKHOLM – XNK Therapeutics AB (“XNK”) today announced that it is entering into a joint Phase II clinical study to treat patients with multiple myeloma using XNK’s leading drug candidate in combination with Sanofi’s anti-CD38 antibody Sarclisa (isatuximab). XNK and Sanofi are both collaborative partners within NextGenNK Competence Center coordinated...
MARKHAM, Canada – Astellas Pharma Canada, Inc. announced that Health Canada has approved a supplemental New Drug Application for XTANDI® (enzalutamide) in a new prostate cancer treatment setting. With this approval, XTANDI becomes the first and only androgen receptor pathway inhibitor (ARPI) approved for use with or without a GnRH...