Latest News

CAMBRIDGE, Mass. – Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive results from Part A of the MOMENTUM study (Study 5051-201), a global, Phase 2, multi-ascending dose clinical trial of SRP-5051, its next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with...
CAMBRIDGE, Mass. – Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared new  of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the Company’s investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). In the first look at expression data from biopsies taken two years after a single administration...
CLINUVEL today announced that it has reached agreement with clinical and academic experts to expand its DNA Repair Program to patients diagnosed with xeroderma pigmentosum-variant (XP-V). The Program commenced in 2020, evaluating SCENESSE® (afamelanotide 16mg) in XP-C patients.“The progression of making afamelanotide available to a further group of XP patients, who...
CAMBRIDGE, Mass. – Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the United States Patent Office (USPTO) issued U.S. Patent No. 10,946,036 with an expiry of June 2037. The claims of...
CAMBRIDGE, Mass. – Scholar Rock (NASDAQ:SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced positive top-line data from the TOPAZ Phase 2 clinical trial evaluating apitegromab (SRK-015) in patients with Type 2 and Type 3 spinal muscular...
CAMBRIDGE, Mass. – Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that findings from its Phase 1 trial evaluating the safety and pharmacologic profile of apitemograb in healthy volunteers were published in the...
LOS ANGELES – Science 37, the industry leader in decentralized clinical trials, announced today a partnership with Xperiome, a global rare disease healthtech company. The partnership further strengthens Science 37’s capabilities to bring research directly to patients, both by easing the patient burden and enabling treating physicians the opportunity to...
Your immune system plays an important function in your health — it protects you against viruses, bacteria, and other toxins that can cause disease. In autoinflammatory diseases, however, the immune system goes awry, causing unprovoked and dangerous inflammation. Now, researchers from the National Institute of Arthritis and Musculoskeletal and Skin...
Bats spread the Ebola virus to humans and play a pivotal role in disease outbreaks, evidence suggests. Ebola, a filovirus, causes fever, vomiting, diarrhoea and sometimes bleeding. There is no treatment or vaccine and 25–90 per cent of infected people die. The virus is transmitted by direct contact with infected...
HOUSTON – A rare, deadly developmental disorder of the lungs called alveolar capillary dysplasia with misalignment of pulmonary veins (ACD/MPV) that usually kills the infants born with it within the first month of life results from deletions or mutations in the FOXF1 transcription factor gene, said a consortium of researchers...