RDR Alert®

Randomized, placebo-controlled trial found a 50% reduction in absolute eosinophil count in 90% of patients.

About 1000 people in the United States have RPE65 gene mutations and inherited retinal diseases.

The investigational anti-fibrotic immunomodulator is being investigated for respiratory, oncology, hepatology, and nephrology indications.

Investigators studied patients from 5 unrelated families to determine the genetic mutations causing the condition.

Research into a rare neurological disorder may help researchers working on more common diseases including autism and epilepsy.

Both entrectinib and polatuzumab vedotin target hard-to-treat cancers.

Female sex on its own was a risk for arrhythmic events, but only for certain LQTS genotypes.

Investigators have found that mice with ALS given the novel treatment showed significant improvements in their cognitive and motor performance.

MPN— myeloproliferative neoplasm—involves the production of too many red blood cells, white blood cells, or platelets in the body.

Providers and patients now have the option to choose a first dose of daratumumab in 1 or 2 infusions.

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).