FORT LEE, N.J. — Kedrion Biopharma is raising awareness for Plasminogen Deficiency Type 1 (PLGD‑1), a rare genetic disorder, through a series of global virtual and in‑person events held in recognition of Plasminogen Deficiency Awareness Day on May 4. PLGD‑1 is an ultra‑rare condition affecting approximately 1.6 per 1 million people...
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FORT LEE, N.J. — Kedrion is proud to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to investigational treatment for Congenital Aceruloplasminemia, a rare genetic disorder affecting iron metabolism. This designation marks a significant milestone in Kedrion’s commitment to addressing the unmet medical needs of patients living with ultra-rare...
A Keele researcher will embark on a two-year study to identify affordable treatments to help children living with spinal muscular atrophy. Dr Melissa Bowerman, of Keele University’s School of Medicine, has been awarded £99,959 by the Academy of Medical Science’s Springboard grant scheme to continue her research into treatments for...
DUBLIN, Ireland — Keenova Therapeutics plc announced the publication of a new manuscript presenting real-world evidence on treatment patterns for patients with plantar fibromatosis. The peer‑reviewed article appears in The Journal of Foot and Ankle Surgery, available here. Why It Matters Plantar fibromatosis is a progressive condition with no FDA-approved nonsurgical treatment options. With...
DUBLIN, Ireland — Keenova Therapeutics plc announced today that new and final updated data from its dedicated ventral curvature analysis of Peyronie’s disease and XIAFLEX® (collagenase clostridium histolyticum) will be shared at the American Urological Association (AUA) annual meeting, taking place May 15–18, 2026. The data to be presented will highlight that the...
PEA RIDGE – Kyler Kasper is living on borrowed blood. He has not yet celebrated his second birthday, but Kyler’s parents expect him to reach it, thanks to blood donations from strangers. Kyler was recently diagnosed with Diamond Blackfan anemia, a rare blood disorder that affects about 500 people in...
Brussels, Belgium – Patients are central to the rare disease community on all levels. This sentiment was echoed by representatives from all stakeholder groups at the 2023 World Orphan Drugs Congress which took place in early November 2023 in Barcelona. The Parliament joined the Alexion – AstraZeneca Rare Disease team...
BOSTON, Mass. — Kelonia Therapeutics, Inc., a clinical-stage biotechnology company pioneering in vivo gene delivery, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for KLN-1010 for the treatment of patients with relapsed and refractory multiple myeloma. KLN-1010 is a novel in vivo gene therapy...
BALTIMORE, Maryland — Researchers at Kennedy Krieger Institute are sharing their expertise on autism spectrum disorder in a medical journal reaching thousands of pediatric professionals worldwide. The journal, Pediatric Clinics, provides the latest clinical information on health and related issues for children and adolescents. The newly released volume is titled...
LEXINGTON, Mass. — Keros Therapeutics, Inc. (Keros) (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta (TGF-ß) family of proteins, today announced the U.S. Food and...