In Historic Move, Israeli Insurer to Cover Cost of Zolgensma for 2 SMA Babies
In an unprecedented decision reached Sept. 26 on the eve of Rosh Hashana — the Jewish New Year — Israel’s…
Articles by Larry Luxner
NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a…
At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo…
Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have.
Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second…
European Initiative Targets Diagnosis, Treatment of Rare Diseases
A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis…
NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet…
European SMA Advocates Question EU’s Willingness to Reimburse for Zolgensma
Now that the U.S. Food and Drug Administration has given its blessing to the AveXis gene therapy Zolgensma…
Zolgensma’s Arrival Adds Urgency to SMA Newborn Screening Efforts in US, Europe
[Editor’s note: This is the fifth in a series of articles on Zolgensma and treatments for SMA, the issues they…
Pennsylvania SMA Patient: ‘Spinraza Has Changed My Life’
Michael Noon, 42, says the thing he hates most about spinal muscular atrophy (SMA) is having to constantly ask…