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Taylor Sabky spent this past Mother’s Day in shock, absorbing the news that her toddler, Purnell, was dying. He’d been diagnosed days before with Niemann-Pick type A — an ultra-rare genetic disease that typically kills children by age 3. It was inconceivable...Rare diseases are increasingly attractive to biopharma companies, which can charge premium prices if they come up with a therapy. (One drug that just hit the market is priced at $750,000 for the first year of treatment.) But before they’ll invest heavily in a field, companies want to see compelling early-stage science. (Keshavan, 6/30)

In this city on the east bank of the Mississippi River, Rebekah Gee, Louisiana’s health secretary, presides over what she calls the “public-health-crisis cradle” of America. Poverty and poor health collide here to produce some of the nation’s worst rates of obesity, premature birth and other maladies. Those problems are deep-rooted and hard to solve. The easy one, at least in theory, is hepatitis C, a liver-damaging virus frequently contracted by injection-drug users that can cause cirrhosis and cancer. (Johnson, 7/3)

Donald Jones used to pay at least $500 a month for a brand-name drug, Gleevec, that’s kept his leukemia at bay for five years. Lately, he’s been paying almost as much for a generic version of the same pill. It’s not supposed to work that way. For decades in the U.S., generic drugs have been cheap, effective alternatives to expensive brand-name treatments. That’s changing with drugs like Gleevec, Novartis AG’s household-name cancer treatment. Generic forms of the drug can cost $150 or more a pill. (Langreth, 6/30)

Adecade ago, a big push began to force drug makers to disclose payments to doctors for speaking, consulting, travel, and research. The campaign was controversial, but reflected concerns that medical practice and research may be unduly influenced by financial ties, and it ultimately led to the creation of a federal database that collects industry payments. Now, though, a group of researchers sought to quantify the extent to which such efforts may have had on actual prescribing. And they found a drop in prescriptions for three types of widely used medicines in Massachusetts, a state that was among the first to require companies to report payments. The law went into effect in July 2009, a few years before the OpenPayments federal database. (Silverman, 6/29)

Federal prosecutors Monday asked a judge to muzzle so-called "Pharma Bro" Martin Shkreli for the balance of his ongoing conspiracy and fraud trial. Raising concern that Shkreli's in-person and social media criticism and statements about the case risk "tainting the jury," prosecutors asked U.S. District Court Judge Kiyo Matsumoto to limit Shkreli's statements outside the Brooklyn courtroom. (McCoy, 7/3)

Two drug companies raised the price of cancer drugs last week for the second time this year. Amgen Inc. raised the price of its Blincyto by 3.9% on Wednesday, according to Bernstein analyst Ronny Gal, and Teva Pharmaceutical Industries Ltd. raised the price of its Treanda by 4.5% on Saturday. Both Blincyto and Treanda are leukemia drugs, while Treanda is also approved for non-Hodgkin lymphoma. (Court, 7/3)

Soaring costs for dozens of common drugs are forcing Minnesotans to skip or skimp on their medications, causing alarm among doctors who say that the price of prescriptions has become a chronic health problem in and of itself. The cost of doxycycline, a generic antibiotic, rose from 24 cents per unit in 2011 to $2.21 cents in 2015, while the cost of Avonex, a multiple sclerosis drug, increased from $778 to $5,129 per unit, according to a Star Tribune review of the latest Medicare Part D drug spending data. (Olson, 7/1)

The Massachusetts Department of Public Health is preparing a pilot program to screen newborns for a rare but often fatal disease: spinal muscular atrophy, or SMA. Last month, the Missouri Legislature approved a bill directing health officials to screen newborns statewide for the disorder. (Zimmerman, 6/29)

Now that the Food and Drug Administration plans to fast-track generic drug applications in hopes of spurring competition and lowering medicines costs, a key question should be asked: Can the agency follow through? A new government report suggests that — yes, the FDA does have the wherewithal. Thanks to a program in which industry pays fees so the FDA can more readily review drugs and inspect plans, the agency has already been reviewing generic drug applications at a faster clip. In fiscal year 2012, the agency spent 28 months on reviews, but that fell to 14 months by fiscal year 2015, according to a newly released report by the U.S. Government Accountability Office. (Silverman, 6/28)