News

June 20, 2018June 20, 2018

Two Mutations Seen to Help Identify SMA Carriers, Allowing Better Family Counseling

Two specific mutations may help in identifying people who are carriers of spinal muscular atrophy (SMA) — those who do not have the disease but can pass it to ... Read more

June 18, 2018June 18, 2018

Nerve Cell Function Impacted When Movement is Limited, Mouse Study Suggests

Neurological health depends as much on signals sent by the body’s leg muscles to the brain as it does on those sent from the brain to the muscles, according ... Read more

June 15, 2018June 15, 2018

#CureSMA2018 – Exicure’s SNA Formulation of Nusinersen Raises SMN Protein Levels with Lesser Toxicity, Mouse Study Reports

Exicure’s gene c prolongs survival, increases the amount of SMN proteins, and shows limited toxicity in a mouse model of the disease, study data show. The investigational compound is a ... Read more

June 13, 2018June 13, 2018

Decreased Levels of Specific Enzyme Involved in SMA Development in Mouse Study

Decreased levels of an enzyme lead to cellular dysfunction and neuronal degeneration involved in the development of spinal muscular atrophy (SMA), a new mouse study suggests. The study, “Neuronal activity regulates DROSHA ... Read more

June 11, 2018June 11, 2018

Targeting Essential Amino Acids Seen to Ease Muscular Symptoms of SMA in Mouse Study

Targeted therapies or dietary supplements that work to improve the metabolism of certain molecules in muscle cells may slow the progression of spinal muscular atrophy (SMA), results of an ... Read more

June 8, 2018June 8, 2018

AveXis, Maker of SMA Therapy AVXS-101, Expanding Manufacturing Operations to North Carolina

AveXis is expanding its operations to North Carolina, opening up a new manufacturing center in Durham County, North Carolina Gov. Ray Cooper recently announced in a press release. The company ... Read more

June 6, 2018June 6, 2018

Canada Trails US in Patient Access to Rare Disease Therapies, CORD President Says

Canada’s healthcare system is excellent for people with common ailments like diabetes or high blood pressure, but it’s “basically failing the nearly three million Canadians with rare diseases.” So ... Read more

June 6, 2018June 6, 2018

Roche Stops Work on Olesoxime After Disappointing Long-term Results in Phase 2 Trial

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 ... Read more

June 4, 2018June 4, 2018

US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases

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 ... Read more

June 4, 2018June 4, 2018

Phase 1 Trial of SRK-015, Potential SMA Therapy, Completes 1st Dosing, Scholar Rock Reports

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 ... Read more

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