Friedreich's Ataxia
Skyclarys is the first drug ever approved for FA. Nobody is capturing whether it's working between clinic visits.
Biogen's Skyclarys showed a 2.4-point mFARS improvement in the MOXIe trial — but secondary endpoints didn't reach significance, and regulators have flagged evidence gaps in real-world functional benefit. Forma captures what the SKYCLARYS PASS registry cannot: daily, structured, patient-reported data on gait, falls, dexterity, cardiac symptoms, and fatigue that proves whether this drug is changing patients' actual lives.
Forecast
Based on prior Topics logging
Topics
How it works
15 seconds of speech. 25 data points.
Forma turns each FA patient into a continuous data stream. By voice or text, patients and caregivers log gait stability and assistive device use, falls with circumstances and injury, upper limb coordination during daily tasks, speech clarity, cardiac symptoms, fatigue severity, and Skyclarys adherence — in under a minute. No forms, no recall bias. The result is a structured, longitudinal dataset across every FA domain — daily mobility trajectories mapped to assistive device transitions, fall frequency correlated with fatigue and environment, fine motor decline tracked through real tasks like handwriting and phone use, and Skyclarys treatment response visible across weeks rather than assumed from an annual mFARS score.
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Forecast
Based on prior Topics logging
Topics
Structured, coded, longitudinal
- Timing
- Ambulatory duration:3 class periods (~3 hrs)Palpitation duration:~10 minutes
- Location
- Cardiac trigger:Physical education classFine motor context:Art class
- Severity
- Mobility transition:Crutches → wheelchairCardiac symptom:Palpitations (fluttery)Dexterity impact:Dropped phone × 2
- Actions
- Assistive device:Forearm crutches
- Outcome
- Trend:Improving (+1 period vs. last week)
From data to insights
What the data reveals
Daily voice logs compound into actionable insights — for patients managing their condition and for researchers running studies.
For Patients
Is Skyclarys actually doing anything for me?
Since starting Skyclarys 14 weeks ago, your daily gait distance has increased from averaging 800 to 1,200 steps before needing the wheelchair. Falls dropped from 6/month to 3/month. Your fatigue scores haven't changed — still averaging 6.8/10 — but your mobility window (time on crutches before switching to the chair) grew from 2.5 to 4 hours. LFTs from last week are normal.
Gait distance on Skyclarys
Why do I fall more at school than at home?
Your school falls happen 3x more often than home falls. The pattern: 80% occur during class transitions in crowded hallways, usually periods 3-5 when fatigue is highest. Home falls cluster around the bathroom. On days you use the wheelchair for transitions after 2nd period, you have zero hallway falls. Your PT might want to adjust the school mobility plan.
Falls by location
Is my speech getting worse?
Your speech clarity is stable overall — but there's a clear fatigue pattern. Morning logs: friends understand you 90% of the time. After 3 PM: that drops to 60%. On days with fatigue scores above 7, afternoon speech clarity drops another 15%. The trend over 3 months is flat, not declining — your dysarthria isn't progressing, it's fatigue-dependent.
Speech clarity by time of day
Summarize my semester for the neurologist.
Gait: crutches for 4 hours/day, wheelchair remainder. Improving since Skyclarys — was 2.5 hours in September. Falls: 3/month (down from 6 pre-Skyclarys). Hallway transitions are the main trigger. Upper limb: handwriting declining — switched to laptop for essays. Typing stable. Speech: mild dysarthria, fatigue-dependent, not progressing. Cardiac: 4 brief palpitation episodes, all during PE. Skyclarys: 93% adherent, LFTs normal x3. Scoliosis: stable, brace compliant. Fatigue: 6.8/10 average — worst after gym and 5th period.
For Researchers
Does Skyclarys improve daily function beyond what mFARS captures?
Across 34 FA patients on Skyclarys, daily mobility composite scores improved by 18% at 6 months — but annual mFARS change was only 1.8 points (below the 3-point MCID proposed for trials). Daily fall rate declined 42% (mean 5.1 to 2.9/month). The disconnect: mFARS captures neurological examination findings; daily data captures functional translation. 71% of patients showing daily functional improvement would be classified as mFARS non-responders at annual assessment.
Daily function vs. mFARS
Trend
What is the real-world mobility trajectory between annual visits?
Daily logging reveals that assistive device transitions are not gradual — 74% of patients show a discrete step-down event (e.g., crutches → wheelchair full-time) over a 2-3 week window, typically triggered by a fall cluster or illness. Monthly trajectory analysis detects these transitions 5.1 months before they would appear on an annual mFARS. Pre-transition fall rate acceleration (>2 falls/week for 3+ weeks) is the strongest early signal, with 79% sensitivity.
Mobility step-down events
Trend
What cardiac symptoms occur between echocardiograms?
Palpitation episodes average 3.8/month across our cohort — 82% unreported at cardiology visits. Exertional dyspnea is logged on 29% of patient-days but attributed by patients to 'normal FA tiredness.' Daily cardiac logging detected symptom patterns prompting early cardiology referral in 4 of 34 patients (12%), with one new arrhythmia diagnosis. Patients with >6 palpitation episodes/month had 2.8x the rate of echocardiographic progression at 12-month follow-up.
Unreported cardiac symptoms
Trend
Can daily data support Skyclarys HTA and label expansion submissions?
Canadian regulators explicitly flagged evidence gaps for Skyclarys: no data on advanced disease, non-neurologic manifestations, or long-term efficacy. Our daily dataset addresses all three — capturing wheelchair-dependent patients (N=11), cardiac and fatigue domains, and 12+ month trajectories. A daily fall-rate endpoint achieves Cohen's d=0.71 for Skyclarys responders vs. 0.28 for annual mFARS. Simulated HTA dossier using daily functional data meets CADTH's real-world evidence threshold for supplemental submission.
HTA evidence package
Trend
First day back from spring break. Made it through 1st and 2nd period on crutches, then switched to the chair. Hands were shaky in art class — couldn't hold the brush steady. Tired by lunch.
Someone bumped me in the hallway between 3rd and 4th period. Crutch slipped and I went down. Knee is scraped. The aide helped me up. Really embarrassing — everyone was staring. Used the chair the rest of the day.
Took Skyclarys before breakfast like always. Felt nauseous until about 10. Mom says I should try taking it earlier. Knee still sore from Tuesday. PT after school — balance exercises on the mat. Exhausting but my PT says my trunk control is better than last month.
Chill day at home. Played video games — controller is getting harder to use, keep hitting wrong buttons. Tried to help mom cook dinner, dropped a glass. She wasn't mad but I was frustrated. Speech was fine today.
Good morning. Made it through 3 periods on crutches — that's one more than last week. Heart felt fluttery during PE, lasted maybe 10 minutes. Sat out the rest of class. Told the teacher it was nothing but it scared me a little.
Got blood drawn after school for liver tests. Hate needles. School was okay — used the chair for hallway transitions after 2nd period like my PT suggested. Zero falls this week so far. Typing my history essay was slow but I got it done.
Friend's birthday party. Dad drove me. Used the wheelchair the whole time — didn't want to deal with crutches. My speech was bad by 9 PM, everyone kept asking me to repeat stuff. Tired but glad I went.
Was just sitting watching TV and my heart started racing. Lasted about 20 minutes. Mom almost called the cardiologist but it stopped. Felt weird after. Didn't do much the rest of the day. Scoliosis brace is bugging me.
Mom got the liver test results — all normal. Doctor says keep going with Skyclarys. School was actually good today. Made it through almost 4 periods on crutches before switching to the chair. That's my best since starting the drug. PT noticed too.
10 days no falls! My PT said the new hallway plan is working — chair for transitions, crutches in class. Handwriting is still bad but I'm getting faster on the laptop. Mom cried when I told her about the no-falls streak. I think the Skyclarys might actually be helping.
New week. Energy was better today — only really crashed after 5th period. Speech was clear enough for my presentation in English. Teacher said she could understand everything. Still using the hybrid plan at school. Neuro appointment next month — mom's printing out my logs.
Key insight
Over three weeks, Mia's daily logs capture what annual mFARS scoring and the SKYCLARYS PASS registry cannot: the hallway fall on day 2 that led to a mobility plan change (chair for transitions, crutches in class), the cardiac palpitation on day 14 that would have gone unreported until her next echo, and the treatment response arc that took her from 2 periods on crutches to nearly 4 — a functional gain invisible to an annual clinic visit but exactly the evidence Biogen needs to demonstrate real-world Skyclarys benefit. By day 19, she hit 10 consecutive fall-free days, a milestone her neurologist can use to justify continued treatment and that Biogen's HTA submissions need to prove the drug is changing patients' daily lives.
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