Dear Congress, The recently enacted federal Right to Try Law (RTT) is really a “Right to Ask” law. And, for the pharmaceutical companies, it’s a “Right to Say No.” Which they certainly almost always will. Case in point: BrainStorm Cell Therapeutics pre-emptively announced that NurOwn, its ALS stem cell treatment candidate,…

BrainStorm Cell Therapeutics has partnered with the Connell and O’Reilly Families Cell Manipulation Core Facility at Dana-Farber Cancer Institute for the manufacturing of NurOwn, a therapy candidate for amyotrophic lateral sclerosis (ALS), for BrainStorm’s ongoing Phase 3 trial. This is the second U.S. production site…

Over the past year, I have become an expert in pain — something I never wanted to be. However, daily severe muscle spasms, cramping, one surgery, and an emergency room visit later, here I am. Despite my plethora of pain medications, I still suffered. Then, I realized that…

ALS has been a blessing to me. You read that right — a blessing. Oh, it has been many other things: ordeal, handicap, curse, millstone, scene-stealer, tormentor, humiliator, predator, interloper, income garnisher, bully, danger, satirist, infidel, and terrorist. It strives, ultimately, to be my terminator. But, if not for ALS,…

Canada’s healthcare system is excellent for people with common ailments like diabetes or high blood pressure, but it’s “basically failing the nearly three million Canadians with rare diseases.” So says Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders (CORD), a Toronto-based network representing 102 patient advocacy…

As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…

AB Science has decided not to seek the re-examination it initially requested after the European Medicines Agency (EMA) issued a negative opinion on marketing authorization for masitinib, the company’s investigational amyotrophic lateral sclerosis (ALS) therapy. The company announced in a press release that the re-examination procedure…

Should scientists have the right to edit the genes of future generations to eliminate hundreds, if not thousands, of potential rare diseases? Or should researchers restrict their use of genome editing to somatic cells, so that they don’t pass changes on to the next generation? What about stem-cell research, three-parent…

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…

Two years after approving it, the 28-member European Union will begin enforcing its General Data Protection Regulation (GDPR) — a tough new law that aims to protect the EU’s 512 million citizens, including rare disease patients, from having their medical records misused, sold, or subject to extortion by hackers, third…