Bionews’ Patient Forums Foster Community and Raise Awareness
Online forums have markedly altered how patients, caregivers, and healthcare professionals communicate and connect. Even when miles apart,…
Articles by Mary Chapman
’31 Days of SMA’ Among Highlights of August, SMA Awareness Month
SMA News Today, having launched forums and added regular columnists, is now running a month-long series to coincide with Spinal…
AveXis’ OneGene Program to Smooth the Path for Families Wanting Zolgensma
More than 90% of rare diseases have no federally sanctioned treatment. So when news came of an approved…
SMA Patients in Saskatchewan, Canada, Gain Wider Access to Spinraza
The Canadian province of Saskatchewan has joined that of Quebec in granting access to Spinraza (nusinersen) therapy to a wider range…
Spinraza Developer Disappointed with Canadian Agency’s Updated Recommendation
While the Canadian Association of Drugs and Technologies in Health (CADTH) recommended that the spinal muscular atrophy treatment Spinraza…
China Approves SMA Treatment Spinraza for Most Patients
Spinraza (nusinersen) has been approved by China’s National Medical Product Administration (NMPA) for the treatment of the most common form…
British MP Pushes to Make Spinraza Treatment Available for SMA Patients
Citing the deteriorating health of a child with spinal muscular atrophy (SMA), a member of the British Parliament is…
Scotland Opens Spinraza to All SMA Patients Using Its Public Health System
Scotland has joined the growing roster of countries making the spinal muscular atrophy (SMA) therapy Spinraza available to patients…
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about…
FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
The U.S. Food and Drug Administration (FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases,…