Announcements

Spinal Muscular Atrophy (SMA) – Update related to the infusions of Spinraza

Following the Press Release of April 7, 2020 regarding the postponement of some injections of the drug Spinraza in patients with Spinal...

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Announcements, Featured

EURORDIS – Rare Barometer, patients with rare diseases share their experiences in COVID 19

A new rare barometer survey related to patient experiences in COVID 19...

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Announcements

BIOGEN PLANS TO INITIATE PHASE 4 STUDY EVALUATING BENEFIT OF SPINRAZA® (NUSINERSEN) IN PATIENTS TREATED WITH ZOLGENSMA® (ONASEMNOGENE ABEPARVOVEC)

BIOGEN PLANS TO INITIATE PHASE 4 STUDY EVALUATING BENEFIT OF SPINRAZA® (NUSINERSEN) IN PATIENTS TREATED WITH ZOLGENSMA® (ONASEMNOGENE...

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Announcements

Positive result announcement from HOPE – 2 clinical trial for Duchenne Muscular Dystrophy by Capricor Pharmaceuticals

Positive result announcement from HOPE – 2 clinical trial for Duchenne Muscular Dystrophy by Capricor Pharmaceuticals   One year...

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Announcements

EMA’s Approval for Gene Replacement Therapy Zolgensma, for SMA patients

On 19/5/2020 AVEXIS announced that “Zolgensma gene therapy received conditional approval from EMA for patients with Spinal Muscular...

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Important information for patients with SPINAL MUSCULAR ATROPHY (SMA)

2/4/2020 Press Release INFORMATION ABOUT PATIENTS WITH SPINAL MUSCULAR ATROPHY (SMA) It is with great pleasure that we announce the arrival...

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Announcements, Featured

Eurordis – Rare Diseases and COVID-19

Rare Diseases community and Eurordis raises alert over discrimination in critical care guidelines during COVID-19 pandemic and urges...

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Announcements, Featured

W.M.S – World Muscle Society, COVID-19 Update 02-04-2020

Amendments to the original document text – Update 02-04-2020  Covid-19 and people with neuromuscular disorders:  World Muscle...

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Announcements, Featured

W.M.S. – World Muscle Society, COVID-19

Covid-19 and people with neuromuscular disorders: World Muscle Society position and advice...

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