Roche has stopped the development of olesoxime, a treatment candidate for spinal muscular atrophy (SMA) following disappointing 18-month results in an ongoing clinical study, the company announced. In a statement shared online through TreatSMA, Roche mentioned the most recent results of the OLEOS Phase 2 trial (…
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…
Scholar Rock announced it has completed dosing a first group of healthy volunteers in its Phase 1 clinical study of SRK-015, a spinal muscular atrophy (SMA) treatment candidate. SRK-105 selectively blocks the activation of myostatin, a growth factor produced primarily in skeletal muscle cells that helps to maintain a healthy muscle…
Envisagenics announced that it has been given a two-year, $1.5 million grant from the National Institutes of Health (NIH) to further advance SpliceCore, a new biomarker and drug discovery platform. The award, a Small Business Innovation Research (SBIR) Phase 2 grant, will help Envisagenics in further developing SpliceCore, a…
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…
A program offered through a partnership between Invitae Corporation and Biogen offers free genetic testing to infants, children and others with clinically diagnosed spinal muscular atrophy (SMA) or suspected of having the disease. The program, SMA Identified, aims to increase access to genetic testing for the SMA community, so…
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…
Cytokinetics and Cure SMA have expanded their existing partnership to increase education, awareness and fundraising for spinal muscular dystrophy (SMA). As a National Platinum Partner for 2018, Cytokinetics will support several of Cure SMA’s initiatives at both local and national levels to help further research of potential…
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 days…
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…
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