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Patients must also receive a brochure and sign risk acknowledgment.

The long-acting, single-dose nerve block can now be used for popularizing procedures such as total shoulder arthroplasty or rotator cuff repair.

FDA grants orphan drug designation to elamipretide for the treatment of Barth syndrome.

At the most recent American Lung Association LUNGFORCE expo, RDR sat down with Albert Rizzo, M.D. of the Christiana Care Pulmonary and Critical Care Medicine Section to discuss previous, current and emerging therapies for idiopathic pulmonary fibrosis.

Upadacitinib (15 mg, once-daily) met both primary endpoints, with 71% of patients achieving ACR20 and 29% achieving clinical remission at week 12.

Researchers found no evidence that adding targeted retinal photocoagulation to ranibizumab treatment improved visual outcomes, decreased treatment burden, or reduced demand for anti-VEGF therapy

This morning, AbbVie revealed positive top-line results from the Phase 3 SELECT-COMPARE clinical trial evaluating upadacitinib in rheumatoid arthritis.

Novartis and AveXis merge spells opportunity for SMA research.

The device is approved for commercial use in more than 50 countries but is limited to investigational use in the United States and Japan.

When taken early in a pregnancy, mifepristone can end a pregnancy that is less than 49 days along. Another notable trait of the drug, though, is its ability to control the high blood sugar in patients with Cushing syndrome who have type 2 diabetes and have either failed surgery or are not candidates for it.

Rare Disease Report tries to keep its audience at the forefront with the latest news from the hematological and oncological spaces. This new infographic provides 5 little-known facts about MPNs that might not necessarily be covered in news articles from RDR or other media outlets.

Rhizen Pharmaceuticals SA's product, Tenalisib (RP6530), is granted orphan drug designation from the FDA for the treatment of CTCL.

How taking time to reflect after the loss of a patient can help physicians endure everyday loss.

Eight weeks of ledipasvir and sofosbuvir for HCV genotype 1 was found as effective as 12 weeks in patients of different race and ethnicity despite guidelines recommending the shorter treatment for non-black patients.

New data has elucidated a mechanism of action by which hormonal replacement therapy could help prevent T2D in women after menopause.

Global studies show the practice is still rare, but growing in countries to have legalized it.

Tarekegn Geberhiwot, MD, of the Institute of Metabolism and Systems Research and colleagues had an article published in the Orphanet Journal of Rare Diseases, outlining the development of clinical management recommendations for Niemann-Pick disease type C.

QurAlis Corporation joins forces with investors MP Healthcare Venture Management (MPH), Amgen Ventures, and Alexandria Venture Investments to find a cure for ALS.

Molecular characteristics that link the genomic profiles of squamous cell carcinomas from 5 areas of the body have been uncovered by a team of researchers supported by the NIH.

Phase 2 Study with Lanifibranor on Non-Alcoholic Fatty Liver Disease in Patients with Type 2 Diabetes Commences.

A stem cell-based implant has been shown in a phase 1/2a trial to not only halt AMD disease progression but possibly improve vision.

The FDA granted orphan drug designation to fluasterone (ST-002) for the treatment of Cushing’s syndrome.

Treatment with nivolumab, followed by ipilumab, produced significantly higher overall survival and objective response rates than sunitinib alone among intermediate- and poor-risk patients with previously untreated advanced renal-cell carcinoma.

A program based out of the Los Angeles County Jail found peer support made a big difference in inmates’ HIV suppression post-release.

Findings suggest that proline and proline-rich metabolites such as trans-4-hydroxyproline are very important for growth during the early stages of C. difficile colonization.

Researchers note that efforts are needed to align clinical practice with clinical guidelines.

The devices have received expanded indication for use in femoral vein closures in addition to previous approval for arterial closures.

Since March 10, almost 100 patients have presented to EDs with vitamin K-dependent coagulopathy, which has been linked to synthetic cannabinoid use.

Researchers Clive Svendsen, PhD and Samuel Sances of the Cedars-Sinai Board of Governors Regenerative Medicine Institute in Los Angeles note that spinal neuron development can be initiated by the human brain’s tiniest blood vessels.

A new assessment of the association between alcoholism and brain structure is emphasized by current data indicating a major increase of 106.7% in alcohol use disorder in the US population aged 65 years or older.