NOVATO, California — The Phase 2b clinical trial will enroll up to six cystinosis patientswith a history of good compliance using the currently availableimmediate-release form of cysteamine bitartrate, the only drug cleared formarketing by the U.S. Food and Drug Administration (“FDA”) and EuropeanMedicines Agency (“EMEA”) to treat cystinosis. The clinical...
treatment News
There are more than 7,000 rare diseases identified in the world today. While “rare” means that less than 200,000 people in the US are affected, to suffer from a rare disease is not actually rare at all. Around 10% of the American population suffers from a rare disease. A shocking...
HRA Pharma Rare Diseases and Celnova Pharma are partnering to meet a major unmet need for rare disease drugs in South America.
Bethesda, Md. — HYFTOR® (sirolimus topical gel) 0.2%, indicated for the treatment of facial angiofibroma associated with tuberous sclerosis complex (TSC) in adults and pediatric patients 6 years of age and older, is now covered by Medicare in Texas, Florida, New York, and California, among other states. Nobelpharma, a pharmaceutical...
Rare diseases are a serious public health concern in India, with an estimated burden of about 80 to 96 million cases reported annually. Moreover, 70-80% of rare diseases are of genetic nature and thus are asymptomatic. Here are their causes, signs, symptoms and treatment
BOSTON, Mass. — Ratio Therapeutics Inc. (Ratio), a pharmaceutical company employing innovative technologies to develop best-in-class radiopharmaceuticals for cancer treatment and monitoring, announced today that dosing of the first cohort has been completed in the ATLAS trial, a Phase 1/2 open-label study evaluating the safety, tolerability, and efficacy of the company’s lead...
BERKELEY, Calif. — Ray Therapeutics (RayTx), a clinical-stage biopharmaceutical company pioneering optogenetic therapies for those living with retinal degenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead candidate, RTx-015, for the treatment of retinitis pigmentosa (RP)....
Dublin, Ireland. Boosting levels of a deficient protein has clear survival benefits for people with the genetic condition alpha-1 antitrypsin deficiency (AATD), according to new research led by RCSI University of Medicine and Health Sciences. The findings underscore a call to make augmentation therapy accessible to patients with AATD in...
Ann Arbor, Mich. – High-grade glioma, an aggressive form of pediatric and adult brain cancer, is challenging to treat given the tumor location, incidence of recurrence and difficulty for drugs to cross the blood-brain barrier. Researchers from the University of Michigan, Dana Farber Cancer Institute and the Medical University of...
New York, NY – In people with pulmonary arterial hypertension (PAH), adding Winrevair (sotatercept-csrk) to standard therapy was linked to longer survival, fewer safety events, and a lower need for lung transplant compared with standard therapy alone, according to new real-world data. “These findings extend clinical trial evidence, suggesting sotatercept [Winrevair] offers meaningful...