RDR Alert®

Investigators examined the interaction between the proteins TSP1 and C7 to look for a potential molecule to treat fibrosis in patients with butterfly syndrome.

Investigators working on Glioblastoma Moon Shot program have made a key discovery on glioblastoma progression, identifying a key target to help stop it.

Cablivi (caplacizumab-yhdp) is the first approved treatment for adults with acquired thrombotic thrombocytopenic purpura (aTTP).

Trazodone is a depression drug, but it blocks the integrated stress response, so it may be a good candidate for ALS.

The oncolytic adenovirus was safe in terms of toxicity in mice models.

Eliot A. Brinton, MD, outlines the continued unmet needs for patients with familial chylomicronemia syndrome (FCS).

The medulloblastoma subset has a poor prognosis, so investigators have been looking for new treatment options.

The FDA has approved Janssen’s ibrutinib plus obinutuzumab for treatment-naïve patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL).

There are only 4 case reports of this cancer in muscular dystrophy patients.

Some of the new subtypes are similar to previously identified ones but show similar prognoses to established subtypes.

The surgery restored arm movement and function in children affected by acute flaccid myelitis.

The FDA has approved trastuzumab-dttb (Ontruzant) for the treatment of patients with HER2-overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma in combination with cisplatin and capecitabine or 5-fluorouracil.

River blindness and elephantiasis are 2 of the leading causes of global disability.

Improvements in muscle and cardiac function were observed for 18 months in tested mice.

The FDA has approved cabozantinib (Cabometyx) for the treatment of patients with hepatocellular carcinoma who previously received sorafenib (Nexavar).

Only patients with very serious, life-threatening conditions with a risk/benefit ratio is acceptable for use of the therapy at this time.

Dipanjan Basu, PhD, is testing non-melanoma drugs, like the investigational therapy omipalisib (GSK2126458) in cultured patient cells.

While the absolute risk of therapy-related blood cancers is low, the treatment is often intensive and linked to substantial morbidity.

In a 10-patient study, sutimlimab halted the destruction of red blood cells in patients with cold agglutinin disease.

The FDA has approved dasatinib (Sprycel) in combination with chemotherapy for the treatment of pediatric patients with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL).

The polio-like AFM is an emerging health concern, with outbreaks documented since 2012.

New research suggests that more healthy people than previously thought have genetic variants associated with disease.

The classic Fabry disease phenotype in men is almost exclusively marked by anti-agalsidase antibody development, despite having no clear clinical impact.

New options for BPDCN and PNH provide patients alternatives to more burdensome standard-of-care treatment.

The FDA has approved pembrolizumab for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC).