Advances

Muscular dystrophy drug launch halts after pricing concernsMarathon Pharmaceuticals is temporarily pausing the launch of its drug to treat a rare form of muscular dystrophy (MD) after concerns over its $89,000 price tag.
New bladder cancer, kidney disease therapies availableFDA recently approved 2 new therapies: an expanded drug indication for locally advanced or metastatic urothelial carcinoma (mUC) and a treatment for secondary hyperparathyroidism (HPT) in adult patients with chronic kidney disease (CKD) on hemodialysis.
The ‘post-truth’ world: How it’s drifting into medicineHenry Rosevear, MD, examines how the political notion of a “post-truth” world has seeped into medicine.
First therapy approved for rare blood cancerFDA recently granted accelerated approved for ibrutinib (Imbruvica, Janssen, Pharmacyclics), the first treatment for patients with a certain type of lymphoma.
Constipation drug wins approvalFDA approved plecanatide (Trulance, Synergy Pharmaceuticals) for adults with chronic idiopathic constipation.
FDA approves hemophilia A drug for childrenFDA recently expanded the use of antihemophilic factor (recombinant), pegylated (Adynovate, Shire plc) for hemophilia A patients under 12 years old.
Advanced ovarian cancer drug approvedFDA recently approved rucaparib (Rubraca, Clovis Oncology) tablets to treat patients with deleterious BRCA mutation (germline and/or somatic)-associated advanced ovarian cancer, who have been treated with 2 or more chemotherapies.
FDA green lights first treatment for rare spinal diseaseFDA approved nusinersen (Spinraza, Biogen), the first drug to treat children and adults with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement.
New diabetes combo drug available soonA new combination drug for type 2 diabetes will available in the US in the coming months.
Study: Compound found in vegetables could have anti-aging effectsA recent study found that the compound, NMN, boosts production of NAD+ in mice and could have anti-aging effects along with additional benefits, but more research is necessary to determine its effect on humans.

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