spinal muscular atrophy

.tb-grid,.tb-grid>.block-editor-inner-blocks>.block-editor-block-list__layout{display:grid;grid-row-gap:25px;grid-column-gap:25px}.tb-grid-item{background:#d38a03;padding:30px}.tb-grid-column{flex-wrap:wrap}.tb-grid-column>*{width:100%}.tb-grid-column.tb-grid-align-top{width:100%;display:flex;align-content:flex-start}.tb-grid-column.tb-grid-align-center{width:100%;display:flex;align-content:center}.tb-grid-column.tb-grid-align-bottom{width:100%;display:flex;align-content:flex-end} .wpv-view-output[data-toolset-views-view-editor="5a9a58ca77cdac89feb80c6ebc580019"] .wpv-collage { grid-column-gap: 15px;grid-row-gap: 15px; } .wpv-view-output[data-toolset-views-view-editor="5a9a58ca77cdac89feb80c6ebc580019"] .wpv-collage > div:nth-child(4n + 1) { grid-area:  auto / auto / span 3 / span 7; } .wpv-view-output[data-toolset-views-view-editor="5a9a58ca77cdac89feb80c6ebc580019"] .wpv-collage > div:nth-child(4n + 2) { grid-area:  auto / auto / span 3 / span 5; } .wpv-view-output[data-toolset-views-view-editor="5a9a58ca77cdac89feb80c6ebc580019"] .wpv-collage > div:nth-child(4n + 3) { grid-area:  auto / auto / span 3 / span 5; } .wpv-view-output[data-toolset-views-view-editor="5a9a58ca77cdac89feb80c6ebc580019"] .wpv-collage > div:nth-child(4n + 4) { grid-area:  auto / auto / span 3 / span 7; } .wpv-pagination-nav-links[data-toolset-views-view-pagination-block="a552fc9653458e0d202e306ac0a1e0e3"] { text-align: left;justify-content: flex-start; } .tb-container .tb-container-inner{width:100%;margin:0 auto} .wp-block-toolset-blocks-container.tb-container[data-toolset-blocks-container="40b05292f70a70284b1c1668151484af"] { background: rgba( 250, 246, 246, 1 );padding: 0px;border: 1px solid rgba( 218, 218, 218, 1 ); } .tb-container .tb-container-inner{width:100%;margin:0 auto} .wp-block-toolset-blocks-container.tb-container[data-toolset-blocks-container="15799f218876513e85a62246af23d50a"] { padding: 15px 20px 10px 20px;margin-top: 0px;border: 0px solid rgba( 0, 0, 0, 1 ); } .tb-button{color:#f1f1f1}.tb-button--left{text-align:left}.tb-button--center{text-align:center}.tb-button--right{text-align:right}.tb-button__link{color:inherit;cursor:pointer;display:inline-block;line-height:100%;text-decoration:none !important;text-align:center;transition:all 0.3s ease}.tb-button__link:hover,.tb-button__link:focus,.tb-button__link:visited{color:inherit}.tb-button__link:hover .tb-button__content,.tb-button__link:focus .tb-button__content,.tb-button__link:visited .tb-button__content{font-family:inherit;font-style:inherit;font-weight:inherit;letter-spacing:inherit;text-decoration:inherit;text-shadow:inherit;text-transform:inherit}.tb-button__content{vertical-align:middle;transition:all 0.3s ease}.tb-button__icon{transition:all 0.3s ease;display:inline-block;vertical-align:middle;font-style:normal !important}.tb-button__icon::before{content:attr(data-font-code);font-weight:normal !important}.tb-button__link{background-color:#444;border-radius:0.3em;font-size:1.3em;margin-bottom:0.76em;padding:0.55em 1.5em 0.55em} .tb-button[data-toolset-blocks-button="d38c7b1e6196431e002ed2a380201dca"] .tb-button__link { background-color: rgba( 207, 46, 46, 1 );font-size: 12px;text-transform: capitalize; } .tb-button[data-toolset-blocks-button="d38c7b1e6196431e002ed2a380201dca"] .tb-button__icon { font-family: dashicons; } .tb-button{color:#f1f1f1}.tb-button--left{text-align:left}.tb-button--center{text-align:center}.tb-button--right{text-align:right}.tb-button__link{color:inherit;cursor:pointer;display:inline-block;line-height:100%;text-decoration:none !important;text-align:center;transition:all 0.3s ease}.tb-button__link:hover,.tb-button__link:focus,.tb-button__link:visited{color:inherit}.tb-button__link:hover .tb-button__content,.tb-button__link:focus .tb-button__content,.tb-button__link:visited .tb-button__content{font-family:inherit;font-style:inherit;font-weight:inherit;letter-spacing:inherit;text-decoration:inherit;text-shadow:inherit;text-transform:inherit}.tb-button__content{vertical-align:middle;transition:all 0.3s ease}.tb-button__icon{transition:all 0.3s ease;display:inline-block;vertical-align:middle;font-style:normal !important}.tb-button__icon::before{content:attr(data-font-code);font-weight:normal !important}.tb-button__link{background-color:#444;border-radius:0.3em;font-size:1.3em;margin-bottom:0.76em;padding:0.55em 1.5em 0.55em} .tb-button[data-toolset-blocks-button="20359d45c52c74fddb37f562d17a0d03"] .tb-button__link { background-color: rgba( 6, 147, 227, 1 );font-size: 12px;text-transform: capitalize; } .tb-button[data-toolset-blocks-button="20359d45c52c74fddb37f562d17a0d03"] .tb-button__icon { font-family: dashicons; } .tb-fields-and-text[data-toolset-blocks-fields-and-text="8d670a655444748b596f1fb0c7115269"] { color: rgba( 0, 0, 0, 1 );margin-bottom: 10px; } .tb-fields-and-text[data-toolset-blocks-fields-and-text="8d670a655444748b596f1fb0c7115269"] p { color: rgba( 0, 0, 0, 1 ); }  .tb-image{position:relative;transition:transform 0.25s ease}.wp-block-image .tb-image.aligncenter{margin-left:auto;margin-right:auto}.tb-image img{max-width:100%;height:auto;width:auto;transition:transform 0.25s ease}.tb-image .tb-image-caption-fit-to-image{display:table}.tb-image .tb-image-caption-fit-to-image .tb-image-caption{display:table-caption;caption-side:bottom} .wp-block-image.tb-image[data-toolset-blocks-image="7d618f72fcf8cf3093933d1e4b7feb11"] { max-width: 100%; } .wp-block-image.tb-image[data-toolset-blocks-image="7d618f72fcf8cf3093933d1e4b7feb11"] img { background-color: rgba( 250, 246, 246, 1 );border: 0px solid rgba( 0, 0, 0, 1 ); } @media only screen and (max-width: 781px) { .tb-grid,.tb-grid>.block-editor-inner-blocks>.block-editor-block-list__layout{display:grid;grid-row-gap:25px;grid-column-gap:25px}.tb-grid-item{background:#d38a03;padding:30px}.tb-grid-column{flex-wrap:wrap}.tb-grid-column>*{width:100%}.tb-grid-column.tb-grid-align-top{width:100%;display:flex;align-content:flex-start}.tb-grid-column.tb-grid-align-center{width:100%;display:flex;align-content:center}.tb-grid-column.tb-grid-align-bottom{width:100%;display:flex;align-content:flex-end} .wpv-view-output[data-toolset-views-view-editor="5a9a58ca77cdac89feb80c6ebc580019"] .wpv-collage > div:nth-child(3n + 1) { grid-area:  auto / auto / span 3 / span 12; } .wpv-view-output[data-toolset-views-view-editor="5a9a58ca77cdac89feb80c6ebc580019"] .wpv-collage > div:nth-child(3n + 2) { grid-area:  auto / auto / span 4 / span 6; } .wpv-view-output[data-toolset-views-view-editor="5a9a58ca77cdac89feb80c6ebc580019"] .wpv-collage > div:nth-child(3n + 3) { grid-area:  auto / auto / span 4 / span 6; } .tb-container .tb-container-inner{width:100%;margin:0 auto}.tb-container .tb-container-inner{width:100%;margin:0 auto}.tb-button{color:#f1f1f1}.tb-button--left{text-align:left}.tb-button--center{text-align:center}.tb-button--right{text-align:right}.tb-button__link{color:inherit;cursor:pointer;display:inline-block;line-height:100%;text-decoration:none !important;text-align:center;transition:all 0.3s ease}.tb-button__link:hover,.tb-button__link:focus,.tb-button__link:visited{color:inherit}.tb-button__link:hover .tb-button__content,.tb-button__link:focus .tb-button__content,.tb-button__link:visited .tb-button__content{font-family:inherit;font-style:inherit;font-weight:inherit;letter-spacing:inherit;text-decoration:inherit;text-shadow:inherit;text-transform:inherit}.tb-button__content{vertical-align:middle;transition:all 0.3s ease}.tb-button__icon{transition:all 0.3s ease;display:inline-block;vertical-align:middle;font-style:normal !important}.tb-button__icon::before{content:attr(data-font-code);font-weight:normal !important}.tb-button__link{background-color:#444;border-radius:0.3em;font-size:1.3em;margin-bottom:0.76em;padding:0.55em 1.5em 0.55em}.tb-button{color:#f1f1f1}.tb-button--left{text-align:left}.tb-button--center{text-align:center}.tb-button--right{text-align:right}.tb-button__link{color:inherit;cursor:pointer;display:inline-block;line-height:100%;text-decoration:none !important;text-align:center;transition:all 0.3s ease}.tb-button__link:hover,.tb-button__link:focus,.tb-button__link:visited{color:inherit}.tb-button__link:hover .tb-button__content,.tb-button__link:focus .tb-button__content,.tb-button__link:visited .tb-button__content{font-family:inherit;font-style:inherit;font-weight:inherit;letter-spacing:inherit;text-decoration:inherit;text-shadow:inherit;text-transform:inherit}.tb-button__content{vertical-align:middle;transition:all 0.3s ease}.tb-button__icon{transition:all 0.3s ease;display:inline-block;vertical-align:middle;font-style:normal !important}.tb-button__icon::before{content:attr(data-font-code);font-weight:normal !important}.tb-button__link{background-color:#444;border-radius:0.3em;font-size:1.3em;margin-bottom:0.76em;padding:0.55em 1.5em 0.55em} .tb-image{position:relative;transition:transform 0.25s ease}.wp-block-image .tb-image.aligncenter{margin-left:auto;margin-right:auto}.tb-image img{max-width:100%;height:auto;width:auto;transition:transform 0.25s ease}.tb-image .tb-image-caption-fit-to-image{display:table}.tb-image .tb-image-caption-fit-to-image .tb-image-caption{display:table-caption;caption-side:bottom} } @media only screen and (max-width: 599px) { .tb-grid,.tb-grid>.block-editor-inner-blocks>.block-editor-block-list__layout{display:grid;grid-row-gap:25px;grid-column-gap:25px}.tb-grid-item{background:#d38a03;padding:30px}.tb-grid-column{flex-wrap:wrap}.tb-grid-column>*{width:100%}.tb-grid-column.tb-grid-align-top{width:100%;display:flex;align-content:flex-start}.tb-grid-column.tb-grid-align-center{width:100%;display:flex;align-content:center}.tb-grid-column.tb-grid-align-bottom{width:100%;display:flex;align-content:flex-end} .wpv-view-output[data-toolset-views-view-editor="5a9a58ca77cdac89feb80c6ebc580019"] .wpv-collage > div:nth-child(1n + 1) { grid-area:  auto / auto / span 6 / span 12; } .tb-container .tb-container-inner{width:100%;margin:0 auto}.tb-container .tb-container-inner{width:100%;margin:0 auto}.tb-button{color:#f1f1f1}.tb-button--left{text-align:left}.tb-button--center{text-align:center}.tb-button--right{text-align:right}.tb-button__link{color:inherit;cursor:pointer;display:inline-block;line-height:100%;text-decoration:none !important;text-align:center;transition:all 0.3s ease}.tb-button__link:hover,.tb-button__link:focus,.tb-button__link:visited{color:inherit}.tb-button__link:hover .tb-button__content,.tb-button__link:focus .tb-button__content,.tb-button__link:visited .tb-button__content{font-family:inherit;font-style:inherit;font-weight:inherit;letter-spacing:inherit;text-decoration:inherit;text-shadow:inherit;text-transform:inherit}.tb-button__content{vertical-align:middle;transition:all 0.3s ease}.tb-button__icon{transition:all 0.3s ease;display:inline-block;vertical-align:middle;font-style:normal !important}.tb-button__icon::before{content:attr(data-font-code);font-weight:normal !important}.tb-button__link{background-color:#444;border-radius:0.3em;font-size:1.3em;margin-bottom:0.76em;padding:0.55em 1.5em 0.55em}.tb-button{color:#f1f1f1}.tb-button--left{text-align:left}.tb-button--center{text-align:center}.tb-button--right{text-align:right}.tb-button__link{color:inherit;cursor:pointer;display:inline-block;line-height:100%;text-decoration:none !important;text-align:center;transition:all 0.3s ease}.tb-button__link:hover,.tb-button__link:focus,.tb-button__link:visited{color:inherit}.tb-button__link:hover .tb-button__content,.tb-button__link:focus .tb-button__content,.tb-button__link:visited .tb-button__content{font-family:inherit;font-style:inherit;font-weight:inherit;letter-spacing:inherit;text-decoration:inherit;text-shadow:inherit;text-transform:inherit}.tb-button__content{vertical-align:middle;transition:all 0.3s ease}.tb-button__icon{transition:all 0.3s ease;display:inline-block;vertical-align:middle;font-style:normal !important}.tb-button__icon::before{content:attr(data-font-code);font-weight:normal !important}.tb-button__link{background-color:#444;border-radius:0.3em;font-size:1.3em;margin-bottom:0.76em;padding:0.55em 1.5em 0.55em} .tb-image{position:relative;transition:transform 0.25s ease}.wp-block-image .tb-image.aligncenter{margin-left:auto;margin-right:auto}.tb-image img{max-width:100%;height:auto;width:auto;transition:transform 0.25s ease}.tb-image .tb-image-caption-fit-to-image{display:table}.tb-image .tb-image-caption-fit-to-image .tb-image-caption{display:table-caption;caption-side:bottom} } 

Five Facts about spinal muscular atrophy

January 28, 2026
Medical Communications Five Facts, Neurology, early diagnosis, five types, gene therapy, muscle weakness, spinal muscular atrophy

1 Spinal muscular atrophy (SMA) is a genetic disease involving the loss of motor neurons in the spinal cord.1 Its …

Feature

Spike in searches relating to spinal muscular atrophy following Jesy Nelson disclosure

January 19, 2026
Medical Education Neurology, spinal muscular atrophy

New research from the Centre for Surgery has shown a significant rise in Google searches relating to spinal muscular atrophy …

News

Biogen’s high-dose Spinraza regimen receives European Commission approval for spinal muscular atrophy

January 19, 2026
Research and Development Biogen, Neurology, Spinraza, spinal muscular atrophy

Biogen’s Spinraza (nusinersen) has received approval from the European Commission (EC) as a new high-dose regimen for the treatment of …

News

Novartis shares new data for Zolgensma in children with SMA

March 5, 2024
Musculo-skeletal disorder, Novartis, Zolgensma, sma, spinal muscular atrophy

Novartis has announced new data to continue the support of the clinical benefits of Zolgensma (onasmenogene abeparvovec), for the treatment …

News

NMD Pharma begins phase 2 trial for spinal muscular atrophy treatment

September 27, 2023
Research and Development Musculo-skeletal disorder, NMD Pharma, clinical trial, spinal muscular atrophy

NMD Pharma has announced that the first patient has been dosed in its phase 2 clinical trial for its CIC-1 …

News

EC approves Roche’s Evrysdi for babies under two months with SMA

August 30, 2023
Research and Development European Commission, Evrysdi, Musculo-skeletal disorder, Roche, spinal muscular atrophy

Roche has announced that the European Commission (EC) has approved the extension of Evrysdi’s marketing authorisation in the EU to …

News

Roche’s Evrysdi drug for SMA shown to significantly increase survival

April 15, 2021
PTC Therapeutics, Roche, pharma, pharma news, spinal muscular atrophy

Roche’s Evrysdi therapy for the treatment of children with type 1 spinal muscular atrophy (SMA) has been shown to increase …

News

Roche’s SMA treatment Evrysdi approved by European Commission

March 30, 2021
Manufacturing and Production EMA, European Commission, PTC Therapeutics, Roche, sma, spinal muscular atrophy

The European Commission has granted marketing authorisation to Roche and PTC Therapeutics’ Evrysdi, for the treatment of patients aged 2 …

News

Roche and PTC’s SMA treatment shows positive trial results

March 16, 2021
Manufacturing and Production PTC, PTC Therapeutics, Roche, sma, spinal muscular atrophy

Roche and PTC Therapeutics’s Evrysdi treatment for children and adults with Type 2 or Type 3 spinal muscular atrophy (SMA) …

News

NICE recommends £1.79m gene therapy for spinal muscular atrophy in babies

March 8, 2021
Sales and Marketing NICE, spinal muscular atrophy

NICE is set to approve a new and potentially curative one-off gene therapy for babies with the rare genetic disorder …

News

Novartis unveils strong Phase 3 efficacy for one-time therapy Zolgensma in paediatric spinal muscular atrophy

October 1, 2020
Research and Development Novartis, Zolgensma, spinal muscular atrophy

Novartis has lifted the curtain on new Phase 3 data from an ongoing trial of 33 participants on the efficacy …

News

Genentech’s Evrysdi becomes first FDA-approved oral treatment for spinal muscular atrophy

August 10, 2020
Sales and Marketing Evrysdi, FDA, Genentech, Roche, spinal muscular atrophy

Genentech’s Evrysdi (risdiplam) has secured FDA approval for the treatment of spinal muscular atrophy (SMA), making it the first oral therapy …

News

Novartis reveals hard-hitting five-year data for its gene therapy Zolgensma in spinal muscular atrophy

March 25, 2020
Research and Development Novartis, Zolgensma, gene therapy, pharma, spinal muscular atrophy

Novartis has revealed five-year data on its gene therapy Zolgensma, boasting that the one-time infusion demonstrated “rapid, significant and clinically …

News