Either by coincidence or by design, MDA Hellas, the Muscular Dystrophy Association of Greece, is located on Elpidos Street in downtown Athens. Elpidos is Greek for “hope” — and that’s exactly what MDA Hellas offers the many neuromuscular disease patients under its care. Antigone Karras, executive manager of the…
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…
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…
Cure SMA will host the world’s largest regular gathering on spinal muscular atrophy next month in Dallas, bringing together leading SMA researchers and clinicians, as well as families living with the disease. The 2018 Annual Cure SMA Conference is set for June 14-17 at the Hilton Anatole Hotel. Its first three…
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…
If 8-year-old Branden Lim had been born in Australia instead of Malaysia, his parents soon would have no problem obtaining Spinraza (nusinersen) to treat his spinal muscular atrophy (SMA). Australian Health Minister Greg Hunt recently announced that, effective June 1, his ministry would add the Biogen therapy to its Pharmaceutical…
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…
The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…
